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 MOST COMMON PATHOGENS

CASE 75-1

QUESTION 1: B.C., a 41-year-old woman, was admitted to the cancer center for placement of a central IV

catheter for administration of chemotherapy to treat acute nonlymphocytic leukemia in relapse. She was

diagnosed 2 years ago and was treated with cytarabine plus daunorubicin, which resulted in a complete

remission for 33 months. On this admission, she will be treated with high-dose cytarabine plus mitoxantrone for

reinduction. What are the most likely pathogens to cause infection in patients like B.C. during periods of

chemotherapy-induced neutropenia?

Bacteria are the primary pathogens associated with infection in febrile neutropenic

patients, especially those occurring early.

16 Bacteremia (reported in approximately

25% of febrile neutropenic patients) is most often caused by aerobic gram-negative

bacilli (including Pseudomonas aeruginosa, Escherichia coli, and Klebsiella

pneumoniae) or aerobic gram-positive cocci (i.e., coagulase-negative staphylococci,

S. aureus, enterococci, viridans streptococci).

17 Since the mid-1990s, the proportion

of gram-negative infections has decreased with a proportional increase in grampositive infections.

2

,

18 Gram-positive bacteria now account for approximately 60%

to 70% of microbiologically documented infections in neutropenic cancer patients.

2

,

19

This is likely attributable (in part) to the frequent use of indwelling IV catheters,

intensive chemotherapy, and widespread use of broad-spectrum antibiotics.

S. aureus (including MRSA) and coagulase-negative staphylococci, streptococci

(including S. pneumoniae and viridans streptococci), and Corynebacterium species

are increasingly important pathogens.

19 Moreover, enterococcal infections (including

vancomycin-resistant enterococci [VRE]) are increasing in frequency. Meningitis

caused by the intracellular organism Listeria monocytogenes can be observed in

patients with defective cellular immunity caused by disease or prolonged

corticosteroid use. In general, anaerobic bacteria are an

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p. 1556

infrequent cause of infection in granulocytopenic patients with hematologic

malignancies.

20 However, they should be suspected in patients with GI malignancies

or with significant disruption of the GI tract.

20 Clostridium difficile is another

anaerobic pathogen that may cause infection in this population.

Pneumocystis jiroveci (formerly known as Pneumocystis carinii or PCP) is a

pathogen responsible for lung infections primarily in patients with HIV infection.

However, PCP can also be responsible for lung infections in some cancer

patients.

21

,

22 Seen predominantly in patients with solid tumors or hematologic

malignancies receiving long-term corticosteroids, PCP may present as subacute,

febrile, hypoxemic, and diffuse pulmonary involvement.

21

,

22

Invasive fungal infections (IFIs) are a major cause of morbidity and mortality

among neutropenic cancer patients and patients undergoing HSCT.

23

,

24 The incidence

of invasive fungal infections in febrile neutropenic patients varies widely because of

differences in definitions, methods of detection, patient populations, and prior use of

antifungal prophylaxis. In general, patients with hematologic malignancies have a

higher incidence of fungal infections than those with solid tumors.

25 Similar to the

risk of bacterial infections, the risk of invasive fungal infections is also related to the

degree and duration of neutropenia. IFIs tend to occur later in the illness. Patients

with prolonged neutropenia (>7 days) or with acute myelogenous leukemia (AML)

undergoing intensive induction therapy, allogeneic HSCT recipients, and those

undergoing therapy for GVHD are at increased risk of acquiring systemic fungal

infections.

23

,

24 Up to 50% of patients who die during prolonged periods of

neutropenia have evidence of deep-seated mycoses.

23 Before the use of fluconazole

prophylaxis in selected populations, Candida species were responsible for most

invasive fungal infections. Most fungal infections in neutropenic cancer patients are

caused by Candida and Aspergillus species.

26–28 Other less common but important

pathogenic fungi are those associated with zygomycosis (e.g., Mucor and Rhizopus

species) and other emerging pathogens (non-albicans Candida, Trichosporon

beigelii, Malassezia species, Cryptococcus neoformans, and Fusarium species).

26–28

Today, invasive infections attributable to Aspergillus species and other molds are a

major cause of IFI-related death (particularly in those with prolonged neutropenia

and GVHD).

23

,

29 Recent reports of improved survival from IFIs are likely related to

newer prophylactic and treatment options and advances in cancer chemotherapy.

30

As previously stated, most viral infections in neutropenic cancer patients are

caused by a reactivation of latent infection rather than new infection.

2

,

3,9,14,15 These

may include hepatitis B virus (HBV), herpes simplex virus (HSV), and Varicella

zoster virus (VZV). Other viruses, such as CMV, can be either reactivation or newly

acquired during HSCT. The risk of viral reactivation in these patients increases in

patients who are seropositive prior to transplant. Respiratory viruses (e.g.,

respiratory syncytial virus [RSV], influenza, parainfluenza), GI viruses (such as

rotavirus and norovirus), and other seasonal viruses may occasionally cause

infection in this population.

STRATIFICATION FOR RISK OF INFECTION

Risk stratification to identify patients most likely to experience neutropenia has

important implications for decisions about prevention, diagnostic strategies, empiric

therapy (selection, route of administration, duration), and site of care.

2–4,9,14,15,31

In

general, the underlying malignancy, status of disease (i.e., active vs. inactive), degree

and duration of neutropenia, and chemotherapy type impact risk. Patients at highest

risk of complications include those with prolonged (>7 days) and profound (<100

cells/μL) neutropenia or with select comorbidities (hypotension, severe mucositis

interfering with swallowing or causing diarrhea, pneumonia, new-onset abdominal

pain, hepatic or renal insufficiency, or neurologic changes).

3

,

4

In contrast, patients

with shorter (≤7 days) anticipated durations of neutropenia without significant

comorbidities are generally considered at low risk of complications of infection.

3

,

4,32

Patients without fever but exhibiting new signs of infection should also be treated as

high risk. Presenting signs and symptoms, cancer type, chemotherapy regimen,

medical comorbidities, and prior history of febrile neutropenia (especially if severe

or prolonged) should also be considered. Patients with select solid tumors (breast,

lung, colorectal, ovarian) and lymphoma most frequently experience neutropenic

fever. Chemotherapeutic regimens associated with the highest (>20%) incidence of

neutropenia are summarized in detail elsewhere

31

(see Section 17, Neoplastic

Disorders).

The factors impacting risk have been utilized by the National Comprehensive

Cancer Network in assigning infection risk in cancer patients as low, medium, and

high.

3 Other risk assessment tools have been proposed,

3

,

14,15

including the

Multinational Association for Supportive Care in Cancer (MASCC) index.

33 Age of

at least 60 years, presence of hematologic malignancies with a history of prior fungal

infections, severe symptoms (particularly hypotension), inpatient site of care, organ

dysfunction (hepatic and renal), and presence of chronic obstructive pulmonary

disease are important variables that result in a low MASCC score (i.e., <21), and

(consequently) patients with any of these factors are considered at highest risk.

Young patients (<20 years) with solid tumors and no or mild symptoms (including the

absence of hypotension) or organ dysfunction are generally at low risk for

complications.

PROPHYLAXIS AGAINST INFECTION

Infection Control

CASE 75-1, QUESTION 2: Should B.C. receive antimicrobial prophylaxis during the neutropenic period? If

yes, which agents should be used?

Exogenous contamination can be prevented by strict protective isolation of patients

in specially designed rooms that maintain a sterile environment. These laminar

airflow rooms are ventilated with air that is passed through a high-efficiency

particulate air filter, which removes greater than 99% of all particles larger than 3

μm. Total protective isolation is accomplished by strict isolation in conjunction with

the administration of sterile food and water, local skin care, and intensive microbial

surveillance. However, this regimen is burdensome, difficult to accomplish, and

expensive, and is recommended only for high-risk patients (such as allogeneic HSCT

recipients).

4 HSCT recipients and candidates undergoing conditioning therapy should

avoid exposure to plants, flowers, and certain foods (such as uncooked fruits and

vegetables), which increase the risk of exposure to fungi.

9

,

34 Close attention and

adherence to adequate hand-washing procedures is essential. In addition, contact

isolation is advocated in circumstances in which the patient may be colonized or

infected with resistant organisms (such as MRSA, VRE, or multidrug-resistant gramnegative pathogens). Finally, isolating the patients from family or caregivers with

potentially contagious respiratory viral illnesses is advocated.

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p. 1557

Antimicrobial Prophylaxis

Early administration of antibiotics (i.e., antibacterials, antifungals, and antivirals)

during the afebrile, neutropenic period in select high-risk patients may result in a

reduction in the number of febrile episodes and subsequent risk of infection. The

goals of such prophylactic regimens are intended to reduce pathogenic endogenous

microflora or prevent the acquisition of new microorganisms. The potential benefits

of such prophylaxis must outweigh the risks of antibiotic-related adverse effects

including drug interactions, the development of resistance (most notable with

antibacterials), and the potential for superinfection. Use of agents for prophylaxis

(such as a fluoroquinolone for antibacterial prophylaxis) may also preclude the use

of the class as empiric therapy for subsequent suspected or documented infections.

In general, lowest-risk cancer patients (such as those receiving standard

chemotherapy for many solid tumors, and neutropenia anticipated to be <7 days)

should not routinely receive antibacterial and antifungal prophylaxis.

3

,

4 Prophylactic

administration of antivirals in such patients is generally restricted to those with a

history of prior infection (such as HSV). In contrast, allogeneic HSCT recipients and

those with acute leukemia, receipt of alemtuzumab therapy, GVHD requiring highdose steroids, and prolonged (>10 days) neutropenia are at highest risk of infection

and should receive antibacterial, antifungal, and (in select cases) antiviral

prophylaxis.

3

,

4

ANTIFUNGALS

CASE 75-1, QUESTION 3: Should antifungal prophylaxis be used in B.C.? What is the role of hematopoietic

growth factors?

Routine antifungal prophylaxis is not indicated in patients with neutropenia at low

risk of infection. However, select patients are at increased risk of developing

systemic fungal infections.

23 Because of the frequency with which such infections are

encountered, difficulties in establishing a diagnosis, poor response rates in patients

with serious invasive infection who are immunocompromised, and effective

prophylactic strategies are necessary in select intermediate and all high-risk patients.

In patients for whom antifungal prophylaxis is indicated, the choice of the agent

depends largely on the risk of invasive mold infections.

ANTIFUNGAL AGENTS

Nonabsorbable Antifungal Agents

Nonabsorbable antifungal agents, such as oral nystatin,

35

,

36 clotrimazole,

37 and oral

amphotericin B,

38 have been studied. Although oral amphotericin B and clotrimazole

reduce the frequency of oropharyngeal candidiasis, none of these antifungals has a

role as primary prophylaxis of invasive fungal infections.

9

In attempts to enhance

activity while minimizing side effects associated with IV administration, aerosolized

delivery of amphotericin B deoxycholate has been investigated for the prevention of

invasive fungal infections in this patient population,

39 and aerosolized liposomal

amphotericin B has been used in leukemic and HSCT patients.

40 Although promising,

the optimal dose, delivery device, and duration for aerosol administration of

amphotericin B preparations have not yet been determined.

Amphotericin B

The use of systemic antifungals for prophylaxis has been summarized elsewhere.

41

Many of the earlier studies evaluated the prophylactic role of IV amphotericin B.

42–44

In general, use of amphotericin B deoxycholate is limited in this setting due to its

toxicities (i.e., infusion-related reactions, nephrotoxicity, and electrolyte

disturbances) relative to available options for prophylaxis. Consequently,

amphotericin B is generally discouraged for primary prophylaxis in high-risk

patients, unless the patient is unable to receive other mold-active prophylactic agents.

If required, a lipid-based formulation would be preferred, especially in patients at

increased risk of amphotericin B-induced nephrotoxicity.

Systemic Azole Antifungals

Systemic azole antifungals (e.g., itraconazole, fluconazole, voriconazole,

posaconazole) may also be considered in select patients, but differ considerably in

their spectrum of activity, adverse reaction profile, drug interactions, and need for

serum concentration monitoring. The newest member of this class, isavuconazole,

while representing potential advantages over comparator agents, has not yet been

studied in such a setting.

Itraconazole has been well studied as an antifungal prophylaxis. In addition to in

vitro activity against many Candida species (e.g., Candida albicans), itraconazole is

active in vitro against Aspergillus species and reduces systemic Candida

infections.

45–47 Although itraconazole oral solution demonstrates improved

bioavailability over the capsule formulation, it is associated with significant GI

intolerance.

48

,

49

Itraconazole is contraindicated in patients with reduced cardiac

ejection fraction due to its negative inotropic effect. Use of newer azoles (such as

posaconazole and voriconazole) has largely replaced the use of itraconazole for

prophylaxis in patients at increased risk of mold infections (such as those patients

undergoing immunosuppressive therapy for GVHD).

Fluconazole prophylaxis decreases the frequency of both superficial (e.g.,

oropharyngeal candidiasis) and systemic fungal infections in HSCT patients

50

,

51 but

not in patients with leukemia.

7

,

52,53 Fluconazole is available as both oral and IV

formulations, and its oral bioavailability is not significantly influenced by changes in

gastric acidity. The IV formulation enables fluconazole to be administered to

critically ill patients or patients who have difficulty swallowing. Although

fluconazole is useful prophylactically, concern about its lack of reliable in vitro

activity against molds limits its use in highest-risk patients. An increased frequency

of isolation of non-albicans Candida (e.g., Candida krusei, Candida glabrata,

Candida parapsilosis) has also been noted in some institutions.

54

Posaconazole has demonstrated improved survival, a reduction in proven or

probable IFI, and a reduction in invasive aspergillosis when compared with standard

prophylaxis (either itraconazole or fluconazole) for the prevention of fungal infection

in patients undergoing chemotherapy for AML or myelodysplastic syndrome.

55

Posaconazole was also effective prophylaxis in allogeneic HSCT recipients

undergoing therapy for GVHD.

56 Posaconazole is currently available in both oral

(solution and tablet) and parenteral (IV) formulations. The oral solution has been

replaced largely by the tablet formulation due to its improved bioavailability and

reduced dependency on the need for coadministration with high-fat meals for optimal

absorption.

57

,

58 Absorption of oral posaconazole may be reduced in patients with

mucositis and in patients receiving acid-suppressing therapy.

59

In addition, the use of

IV posaconazole is not recommended in patients with significant renal impairment

because of the potential toxicity of the vehicle.

Voriconazole’s use as an antifungal prophylaxis in cancer patients at increased

risk of mold infections is not well supported by clinical data despite its established

use in the treatment of invasive aspergillosis.

60–62 Side effects (most notably

hepatotoxicity, rash, phototoxicity) and the increased potential (relative to other

azoles and the echinocandins) for drug interactions with voriconazole may limit its

prophylactic use to patients at highest risk of mold infections. Similar to IV

posaconazole, the use of IV voriconazole should be avoided in patients with

significant renal impairment because of the potential toxicity of the vehicle. Oral

voriconazole should be administered either 1 hour before or after a meal to optimize

its absorption.

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p. 1558

Echinocandins

The echinocandins (e.g., caspofungin, micafungin, and anidulafungin) may be useful

as a prophylactic strategy in high-risk patients. Micafungin has been compared with

fluconazole in autologous and allogeneic HSCT recipients.

63 Based on a composite

endpoint (which included absence of breakthrough fungal infection and absence of

empiric modifications to the antifungal regimen owing to neutropenic fever),

micafungin was found to be superior. Although breakthrough candidemia, survival,

and adverse events were similar in both groups, a trend toward a reduction in

invasive aspergillosis in the allogeneic HSCT population was noted in the

micafungin group. Micafungin is currently US Food and Drug Administration (FDA)–

approved for the prevention of Candida infections in HSCT patients.

SELECTION AND MONITORING OF PROPHYLACTIC ANTIFUNGALS

The use of primary antifungal prophylaxis in cancer patients with neutropenia should

be reserved for patients at intermediate or high risk of invasive fungal infections

(IFIs).

3

,

4 Patients with acute lymphocytic leukemia receiving remission or salvage

induction chemotherapy are at intermediate risk and should be considered for

prophylaxis. Although autologous HSCT recipients may not routinely benefit from

fungal prophylaxis (most notably those without evidence of mucositis), those with

prolonged neutropenia, mucosal damage, or receipt of purine analogs should receive

primary prophylaxis.

9 Acceptable options for prophylaxis against Candida species

include azoles (e.g., fluconazole, itraconazole, voriconazole, posaconazole) and the

echinocandins (micafungin and caspofungin).

3

,

4 Of these options, fluconazole is the

most commonly used agent. In the setting of colonization with fluconazole-resistant

Candida species (such as C. krusei, C. glabrata), an echinocandin (such as

micafungin) is preferred.

3

,

4,9,63

In contrast, patients with higher risk for mold

infections (such as AML/MDS patients, or those with GVHD receiving intensive

immunosuppressive therapy regardless of neutropenia) should be considered for

prophylaxis with mold-active drugs (such as posaconazole, voriconazole,

echinocandins, or amphotericin B) during periods of risk.

3

,

4,9 Mold-active agents are

also recommended in the setting of anticipated periods of prolonged (at least 2

weeks) neutropenia, or prolonged neutropenia immediately before HSCT.

4

Use of itraconazole, posaconazole, and voriconazole for prevention of fungal

infections should be avoided in patients receiving vinca alkaloids (such as

vincristine) due to their inhibition of the cytochrome P4503A4 (CYP3A4) isoenzyme

and the resulting reduction in drug clearance of the vinca alkaloid. Fluconazole,

while a CYP34A inhibitor, is less potent than these other azoles. Voriconazole also

inhibits other cytochrome P450 isoenzymes to the greatest extent, expanding its

potential for significant drug–drug interactions. The impact of isavuconazole on vinca

alkaloids has not yet been report, but the potential for interactions does exist.

Prophylaxis is generally continued during the period of neutropenia. In those with

acute leukemia, myelodysplastic syndrome (MDS), or autologous HSCT recipients,

prophylaxis should continue until day 75 after transplant or through induction therapy

for patients with leukemia.

64 Patients with a history of documented Aspergillus

infection undergoing intensive chemotherapy should be considered for voriconazole.

9

Although the addition of a second prophylaxis (e.g., echinocandin) may be

considered, the benefits of combination therapy for secondary prophylaxis are

unknown.

Prophylactic use of antifungals may also be used in patients with a prior history of

invasive disease due to Candida spp. or filamentous fungi. Such prevention (known

as secondary prophylaxis) should be considered in such patients during subsequent

chemotherapy or stem cell transplantation for the duration of immunosuppression.

In contrast to the predictable serum concentrations resulting from fluconazole

administration, itraconazole, voriconazole, and posaconazole exhibit significant

variability in drug concentration, most notably following oral administration.

Therefore, serum concentration monitoring of these agents may help assure optimal

drug exposure while reducing the potential for concentration-related toxicities.

However, determination of definitive target concentrations for prophylactic use is

hampered by the lack of controlled, prospective clinical trials. For itraconazole,

steady-state trough concentrations of >0.5 mcg/mL or greater have been

recommended. Voriconazole steady-state serum concentration of >0.5 to 4 mcg/mL

has been recommended for use as prophylaxis. Studies regarding the optimal trough

concentration of posaconazole for prophylaxis differ, but range between 0.5 and 0.7

mcg/mL.

65

,

66

NONABSORBABLE ANTIBACTERIALS

Because the GI tract is an important reservoir of potential pathogens, gut

decontamination has been investigated.

19 However, use of nonabsorbable

antibacterial agents has been replaced by oral, absorbable antibiotics.

3

,

4

ABSORBABLE (SYSTEMIC) ANTIBACTERIALS

Trimethoprim–Sulfamethoxazole

Although trimethoprim–sulfamethoxazole (TMP–SMX) decreases bacterial

infections in neutropenic patients,

67

,

68

it may not reduce mortality in this patient

population. The potential benefits of TMP–SMX prophylaxis must be carefully

balanced against the potential for drug-induced bone marrow suppression,

hypersensitivity reactions, hyperkalemia, nephrotoxicity, pancreatitis, the emergence

of resistant organisms (e.g., E. coli), and the development of superinfections. In

addition, leukemic patients receiving mucotoxic chemotherapy and TMP–SMX

prophylaxis may be at increased risk for infections caused by viridans

streptococci.

69

,

70 Therefore, TMP–SMX should not be routinely used for primary

prophylaxis (except as noted below).

TMP–SMX prevents P. jiroveci (PCP) pneumonia in both ALL and HSCT patient

populations. Patients with malignancy at highest risk for experiencing P. jiroveci

(PCP) pneumonia (i.e., patients with acute lymphocytic leukemia receiving intensive

chemotherapy, those with acquired immunodeficiency syndrome, allogeneic HSCT

recipients, those receiving alemtuzumab, patients with GVHD, and those in whom

neutropenia is anticipated to exceed 10 days) should receive TMP–SMX

prophylaxis.

3

,

9 Recipients of T-cell-depleting agents (e.g., fludarabine or cladribine),

cancer patients receiving prolonged or high-dose corticosteroids (>20 mg of

prednisone or its equivalent daily), and autologous HSCT recipients should also be

considered for prophylaxis with TMP–SMX.

9

In such settings, primary prevention

should be continued for up to 6 months (in the case of HSCT recipients) or longer (in

cases in which immunosuppression is continued). In the setting of alemtuzumab

therapy, such prophylaxis would generally continue for at least 2 months and until the

CD4 count exceeded 200 cells/μL.

3

In patients requiring PCP prophylaxis but unable

to tolerate TMP–SMX, patients should receive alternate prophylaxis with either

atovaquone, dapsone, or pentamidine (either IV or via aerosol).

3

,

9

Fluoroquinolones

The fluoroquinolones ciprofloxacin and levofloxacin are used by some centers as

prophylaxis for adult patients at high risk of infection.

71

,

72 Of concern, however, is

the increasing frequency of gram-positive infections (including viridans streptococci)

and resistant gram-negative bacilli (most notable in P. aeruginosa and E. coli)

73

in

patients receiving fluoroquinolone prophylaxis.

69

,

70,74,75 Meta-analyses report

reductions in mortality in high-risk patients receiving prophylaxis with these

fluoroquinolones.

71

,

76 Because

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p. 1559

fluoroquinolone prophylaxis is offset by the emergence of resistant organisms,

routine prophylactic use in low-risk patients should generally be avoided.

4 However,

those at intermediate or high risk of bacterial infections (i.e., those patients with an

ANC ≤100 neutrophils/μL for >7 days) should be considered for fluoroquinolone

prophylaxis until either the onset of fever (at which time empiric antibacterials

would be started) or resolution of severe neutropenia.

4

,

9 Levofloxacin has been

recommended over ciprofloxacin in patients at increased risk of oral mucositisrelated infection with invasive viridans streptococci.

4 Regardless of the

fluoroquinolone chosen, local resistance patterns should be closely monitored before

such prophylaxis is chosen.

Penicillin

Because of the increased risk of invasive pneumococcal infections, select patient

populations (notably asplenic patients, allogeneic HSCT recipients [owing to

functional asplenia and impaired B-cell immunity], and those undergoing

immunosuppression for GVHD) should be considered for prophylaxis with

penicillin. In patients with chronic GVHD, penicillin prophylaxis may be continued

during the administration of immunosuppressives. In HSCT recipients, prophylaxis

should begin 3 months after transplant and continue for 1 year after transplant.

Alternative prophylaxis should be considered in areas where penicillin resistance in

pneumococci is significant.

ANTIVIRALS

Most HSV disease in cancer patients is a consequence of reactivation from latent

infection. High-risk patients (e.g., seropositive for HSV undergoing allogeneic HSCT

or induction or re-induction therapy for acute leukemia) or patients previously

requiring treatment for HSV reactivation should be given antiviral prophylaxis. Both

oral or IV acyclovir and oral valacyclovir are appropriate initial choices in most

patients. Published data for famciclovir for this indication are lacking. Those

receiving foscarnet or ganciclovir (most commonly for prevention and treatment of

CMV infection) do not require additional prophylaxis for HSV, given the activity of

these against H. simplex. Prophylaxis should be administered during periods of

neutropenia and for at least 1 month after HSCT.

3

,

4 The duration may be extended in

those undergoing allogeneic HSCT with GVHD. Patients receiving alemtuzumab may

also require extended prophylaxis of up to 2 months after completion of therapy or

recovery of CD4

+ cells count >200 cells/μL, whichever is later.

Similar to HSV, VZV in cancer patients is most commonly reactivation. HSCT

patients who are seropositive for VZV should also be given long-term prophylaxis

(6–12 months for autologous HSCT and at least 1 year for allogeneic HSCT

patients),

4 particularly with receipt of either bortezomib or alemtuzumab.

3 Extending

prophylaxis in allogeneic HSCT patients should be considered in the setting of

continued immunosuppressive therapy. Like for HSV, patients receiving alemtuzumab

may also require extended prophylaxis of up to 2 months after completion of therapy

or recovery of CD4+ cells count >200 cells/μl, whichever is later. Agents active

against HSV are also useful for VZV prevention. In one study, low-dose (i.e., 500

mg/day, 3 times weekly) valacyclovir prophylaxis after a 35-day course of IV

acyclovir is safe and effective in allogeneic HSCT recipients.

77

Patients at highest risk of either CMV reactivation or primary infection (e.g.,

allogeneic HSCT patients, those receiving alemtuzumab, and those with GVHD

requiring high-dose steroids) should be considered for antiviral prophylaxis against

this pathogen. One strategy is the administration of all patients at risk (universal

prophylaxis). Given the toxicity of ganciclovir IV, valganciclovir PO, foscarnet IV,

and cidofovir IV (the most potent agents for CMV), a second strategy, known as

preemptive, involves administration of antivirals prior to symptoms by following

serologic evidence (based on CMV pp65 antigen or two consecutive CMV PCR

tests) of viral replication. Foscarnet and cidofovir IV are generally reserved as

second-line agents for preemptive therapy (such as in the setting of neutropenia

secondary to ganciclovir). The potential role of valganciclovir PO (an oral pro-drug

of ganciclovir) as a preemptive strategy in this population has been evaluated and is

now considered a viable option in the absence of GVHD involving the GI tract.

78–82

Finally, a third prevention strategy is to combine use of less effective yet safer agents

(such as acyclovir or valacyclovir) together with active surveillance (up to 6 months

in allogeneic HSCT patients) and, when serologic evidence of virus replication

exists, initiate PO valganciclovir or ganciclovir IV therapy. Surveillance for CMV

may be extended in patients with chronic GVHD receiving immunosuppressive

therapy until recovery of CD4+ count of 100 cells/μL or greater.

Patients with prior infections with hepatitis B virus (HBV) or hepatitis C virus

(HCV) can experience reactivation of infection secondary to immunosuppressive

therapy. Reactivation of infection can occur in patients infected with HBV

undergoing immunosuppressive therapy (most notably in allogeneic HSCT recipients

and those receiving anti-CD20 or anti-CD52 monoclonal antibodies). Therefore,

serologic screening (utilizing testing for hepatitis B surface antigen [HBsAg] and

hepatitis B core antibody [HBcAb]) is generally performed in patients at increased

risk of infection. Prophylaxis should be considered in patients with positive tests.

Patients with one or more of these screening tests positive often undergo further

testing for active viral replication (utilizing a quantitative PCR test for HBV DNA).

Preemptive therapy should be considered in patient with evidence of viral

replication. Despite limited data in cancer patients, the nucleos(t)ide analogs

adefovir and tenofovir have largely replaced lamivudine monotherapy in such

settings due to the high incidence of viral resistance with this strategy. Other agents

considered for HBV prophylaxis may include entecavir and telbivudine.

Respiratory tract infections caused by RSV, influenza, and parainfluenza are less

commonly observed in patients with neutropenia. Although response to influenza

virus vaccine may be attenuated, patients undergoing cancer treatment should receive

annual vaccinations with inactivated influenza vaccine.

4 Whenever possible, the

timing of such vaccinations may be best between cycles (>7 days after or >2 weeks

before next treatment). In contrast, immunocompromised patients should not receive

the intranasal live virus vaccine.

4

Hematopoietic Growth Factors

Hematopoietic colony-stimulating factors (CSFs) such as granulocyte CSF (G-CSF;

filgrastim), pegylated G-CSF (pegfilgrastim), or granulocyte-macrophage CSF (GMCSF; sargramostim) are important adjuncts in cancer patients.

83

,

84 Studies in cancer

patients receiving myelosuppressive or myeloablative chemotherapy have

demonstrated that concurrent use of the CSFs can reduce the duration of neutropenia.

The selection of one CSF agent over another is often based on practitioner preference

rather than clinical data.

Hematopoietic growth factors (more specifically G-CSF [filgrastim] or pegylated

G-CSF [pegfilgrastim]) reduce the risk of chemotherapy-induced febrile

neutropenia.

10

,

31,85–87 Risk factors for neutropenia include age (i.e., >65 years),

medical history (including prior history of febrile neutropenic episodes, nutritional

status, unstable comorbidities, and presence of active infections), disease

characteristics (especially those involving bone marrow resulting in cytopenias), and

myelotoxicity of the regimen (including both chemotherapy and radiation) used to

treat the underlying malignancy.

31

,

85–87 Routine use of CSFs for the prevention of

febrile neutropenia should be discouraged in patients at low risk (<10%) of

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p. 1560

febrile neutropenia.

4

,

31,87

In contrast, guidelines advocate the use as primary

prophylaxis in patients at high risk (>20%) of fever and neutropenia. Some

guidelines also recommend administration of CSFs for primary prevention for

patients >65 years with diffuse aggressive lymphoma treated with curative

chemotherapy (especially in the setting of significant comorbidities and in those

receiving select dose-dense chemotherapy regimens). Although benefits of CSFs are

less clear in patients with intermediate risk (10%–20% risk of febrile neutropenia),

they should be considered on a case-by-case basis.

84 CSFs are generally avoided as

prophylaxis in patients undergoing radiation therapy concomitant with chemotherapy

because of the potential for thrombocytopenia

88 or reductions in tumor response.

89

Administration is generally continued 3 to 4 days after chemotherapy administration

and continued until a sufficient and stable postnadir ANC recovery is established.

10

,

84

Pegylated G-CSF may be preferred in some settings owing to the convenience of

single administration.

31

,

90

Consideration of the use of CSFs as secondary prophylaxis (i.e., prevention of

febrile neutropenia in patients undergoing second or subsequent cycles of

chemotherapy) should be based on repeated assessments of patient risk of febrile

neutropenia.

84 Patients experiencing prior episodes of febrile neutropenia or

neutropenia limiting the dose of chemotherapy without prior CSFs should be

considered for secondary prophylaxis.

84

Other Agents

Although beyond the scope of this chapter, select vaccinations have also been

recommended for both autologous and allogeneic HSCT recipients (primarily caused

by a decline in antibody titers to many vaccine-preventable diseases).

9 These include

(but are not limited to) administration of pneumococcal, influenza virus and H.

influenzae vaccines. Live vaccinations should be avoided in this population.

However, because immune response may be altered immediately after transplant,

delays of up to 3 months after cessation of immunosuppressive chemotherapy are

recommended for many vaccines. In addition, serologic testing of antibody response

is recommended in selected settings.

9

Although data to support use are sparse, IV immunoglobulins have been

recommended for HSCT recipients with severe hypogammaglobulinemia (i.e., serum

immunoglobulin G level <400 mg/dL) and recurrent infections.

9

Intravenous

immunoglobulins (IVIG) have been used as adjuncts in the prevention and treatment

of CMV infections. In contrast, granulocyte transfusions have not been proven to be

effective in either prevention or treatment of infection in the neutropenic cancer

patient.

91

INFECTIONS IN NEUTROPENIC CANCER

PATIENTS

Clinical Signs and Symptoms

CASE 75-1, QUESTION 4: Seven days after completing chemotherapy, B.C. experienced a fever of 102°F

(orally). Vital signs are blood pressure, 109/70 mm Hg; pulse, 102 beats/minute; and respirations, 25

breaths/minute. Physical examination demonstrates a clear oropharynx without exudates or plaques. Chest and

cardiac examination are normal. The exit site for the Hickman catheter is clean and nontender without signs of

erythema or induration. The perineum and rectum are nontender, and no masses are noted. Laboratory data are

as follows:

Hematocrit, 20%

Hemoglobin, 7 g/dL

WBC count, 1,400 cells/μL, with 3% polymorphonuclear leukocytes (PMNs), 1% band forms, 70%

lymphocytes, and 22% monocytes

Platelet count, 17,000 cells/L

Blood glucose, 160 mg/dL

Serum creatinine (SCr), 1.1 mg/dL

Blood urea nitrogen (BUN), 24 mg/dL

What are the signs and symptoms of infection in B.C.? What are the most common sites and sources of

infection in patients such as B.C.?

B.C. has an ANC of 48 cells/μL (1,400 WBCs/μL × [0.03 PMNs + 0.01 bands])

and is therefore at high risk for infection.

Fever in neutropenic patients is defined as a single oral temperature of at least

38.3°C (101°F) or a temperature of at least 38.0°C (100.4°F) for more than 1 hour in

the absence of an obvious cause.

3

,

4

It is the earliest (and often the only) sign of

infection in neutropenic patients, because typical signs can be modified or absent in

this patient population.

4 However, only 48% to 60% of patients with febrile

neutropenia have occult or documented infections.

3 Noninfectious sources of fever in

the neutropenic cancer patient include inflammation, tumor progression, tumor lysis,

adverse drug reactions, and transfusion reactions.

3

,

14,15 Signs and symptoms

consistent with a diagnosis of infection without fever should be considered to be

infection in the neutropenic host until proven otherwise.

3

In patients with documented infections, the most common sites are skin, mouth,

throat, esophagus, sinuses, abdomen, rectum, liver, vascular access, lungs, and

urinary tract.

3

,

92 Although the lung is the most common site of serious infection in

neutropenic cancer patients, fever and dry cough are often the only presenting signs of

pneumonia.

92 The impaired inflammatory response results in scant sputum production,

and sputum Gram stains often contain few PMNs. Radiologic evidence of a

pulmonary infection can be minimal or absent, and the chest examination is frequently

not diagnostic.

93 Pneumonia is associated with high mortality in neutropenic patients,

particularly in the setting of bacteremia. In the presence of shock, a mortality rate of

approximately 80% has been observed in these patients.

94

Invasive procedures such as venipuncture, central IV catheter placement (e.g.,

Hickman catheter), and skin biopsies are associated with cellulitis and systemic

infections. However, the typical signs and symptoms of infection (e.g., pain, heat,

erythema, swelling) are often absent in part owing to inadequate granulocytes.

92

Colonization of these lesions may result in local infection and the potential for

systemic dissemination of bacteria and fungi. Bacteremia occurs primarily from entry

of bacteria through the skin or through unrecognized ulcerations in the GI and

perirectal areas.

Confirmation of Infection

Because of the frequent lack of physical signs and symptoms of infection, the

clinician must obtain an accurate history (including the cancer type and treatment

regimen, new signs of infection, antimicrobial prophylaxis, prior infections, and

comorbidities) and conduct a careful physical examination at the first sign of fever. A

detailed search for subtle signs and symptoms of inflammation at the most common

sites, such as the oropharynx, bone marrow aspiration sites, lung, periodontium, skin,

vascular catheter access sites, nail beds, and perineum (including the anus) is

necessary. Before antibiotics are initiated, two sets of blood cultures (with

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p. 1561

each set consisting of two culture bottles) should be obtained.

3

,

4,14

In patients with

indwelling central venous catheters, one of the two sets of blood cultures should be

obtained from the catheter to help rule out catheter-related infection.

4 Diarrheal

stools may be tested for the presence of C. difficile. Additional Gram stain and

cultures (e.g., stool, urine, skin, IV site, respiratory specimens) should be obtained

depending on signs and symptoms.

4 The yield from such cultures, however, may be

affected by the prior or concurrent administration of prophylaxis.

2 Chest radiographs

and oximetry should be obtained in the presence of respiratory symptoms.

4

Respiratory virus testing should take place for patients with upper respiratory tract

infection symptoms (i.e., coryza) or cough.

4

In settings in which pulmonary

aspergillosis is suspected, further radiologic evaluations (such as computed

tomography scans) should be considered.

29 A complete blood count, serum

electrolytes, coagulation, C-reactive protein, urinalysis, and assessment of organ

function (e.g., liver and kidney function) should be obtained to assist in drug dosing

and monitoring for treatment-related toxicities.

4

Recent advances have been made in nonculture-based diagnostic tests that help

support (or in some cases eliminate) the diagnosis of infection in these patients.

95

Such studies include C-reactive protein

96–98 and procalcitonin.

98–100 However, these

tests are not routinely ordered, and their role in the treatment of the neutropenic

cancer patient has not yet been established.

4 Galactomannan (specific for

aspergillosis)

101 and β-D-glucan testing may assist in the diagnosis of fungal

infections.

102

,

103 Serial galactomannan testing has also been used preemptively to

initiate antifungal therapy before overt signs and symptoms of invasive fungal

infections occur.

104

In addition to other issues regarding sensitivity and specificity of

these assays, both galactomannan and β-D-glucan are affected by prior or current

receipt of antifungals.

29 At present, the use of these tests should be restricted to

persistent fever despite other etiologic investigations.

4

Significance of Colonization

CASE 75-1, QUESTION 5: How can an infection be confirmed in patients such as B.C.?

Several factors influence the colonization and subsequent infection by

microorganisms in cancer patients. Organisms isolated from infected patients can be

found in endogenous flora or acquired during hospitalization.

105 Factors leading to

colonization include staff-to-staff and patient-to-patient transmission (e.g., lack of

frequent and adequate hand hygiene), direct transmission from the environment (e.g.,

inadequately disinfected bathtubs, sinks, toilet bowls), foods (e.g., raw fruits and

vegetables), inhalation from contaminated fomites (e.g., respirators, ventilating

systems), and IV access devices. In addition to immunosuppression, the underlying

malignancy and associated chemotherapy diminish the cancer patient’s resistance to

colonization and infection.

The acquisition of and subsequent colonization by potentially pathogenic microbes

may be detected by serial surveillance cultures of specimens obtained from various

body sites such as the nasopharynx, axilla, urine, and rectum. Such surveillance

cultures may be useful for infection control purposes. However, little clinically

useful information is gained in the absence of infection. Therefore, surveillance

cultures are generally restricted to select patients for infection control purposes. In

such cases, culture of the anterior nares (for MRSA) or rectal samples (for VRE or

multidrug-resistant gram-negative bacilli) may be performed.

In summary, B.C.’s surveillance culture results indicate that she is colonized with

several potential pathogens associated with infection in the immunocompromised

host, but these results are probably not useful in selecting empiric antibiotics for her

fever.

EMPIRIC ANTIBIOTIC THERAPY

Rationale

CASE 75-1, QUESTION 6: Routine surveillance cultures of swabs taken from B.C.’s axillae, nasopharynx,

and rectum grew Corynebacterium jeikeium (axillae), S. aureus (axillae and nasopharynx), and Enterococcus

faecium (rectum). What is the significance of these culture results? Should routine, serial surveillance cultures

be performed in patients such as B.C.?

Neutropenic cancer patient with fever and/or other signs and symptoms of

infection should undergo prompt risk assessment and initiation of antibiotic therapy.

Once cultures are obtained, these patients should be emergently started on broadspectrum antibacterials. Prompt institution should not be delayed if cultures cannot be

obtained. Early studies confirmed high mortality in neutropenic patients with

untreated gram-negative infections up to 24 to 48 hours after the onset of fever. Crude

mortality rates secondary to P. aeruginosa bacteremia approached 91%.

6 Prompt use

of empiric, broad-spectrum antibiotics has resulted in significant reductions in

infectious mortality rates.

Initial Empiric Antibiotic Regimens

CASE 75-1, QUESTION 7: Should B.C. be started on antibiotic therapy immediately? Is this rational in view

of the fact that neither the source of her fever nor the pathogen has been established?

Practice guidelines prepared by the National Comprehensive Cancer Network,

3

the

Infectious Diseases Society of America,

4 and the European Society of Medical

Oncology

14

identify antimicrobial options for the treatment of fever in the neutropenic

cancer patient. The ideal antibiotic regimen for empiric management in this setting

(i.e., those without evidence of site- or pathogen-specific infections) remains

controversial. High-risk patients requiring IV therapy are often candidates for

monotherapy regimens, including an antipseudomonal third-generation cephalosporin

(e.g., ceftazidime), a fourth-generation cephalosporin (e.g., cefepime), or an

antipseudomonal carbapenem (e.g., imipenem–cilastatin or meropenem).

3

,

4,14,15

Initial, empiric use of additional antibiotics (such as fluoroquinolones,

aminoglycosides, and vancomycin) may be added to patients who are clinically

unstable.

4 Alternative initial empiric parenteral regimens (excluding those containing

vancomycin) have been investigated in this patient population.

3

,

4,14,15,34 However,

because no significant differences exist among any of these empiric approaches,

monotherapy is employed in most patients.

Optimal Antibacterial Spectrum

CASE 75-1, QUESTION 8: What pathogen- and patient-specific factors should be considered when initiating

empiric therapy for B.C.?

Despite continued development in antibacterial drugs, the empiric management of

febrile neutropenic patients is complicated by the changing spectrum of bacterial

pathogens and their antimicrobial susceptibilities. Empiric antibiotic regimens

should provide broad-spectrum coverage against the potential gram-negative bacilli

most commonly isolated from neutropenic cancer patients (e.g., E. coli, K.

pneumoniae, P. aeruginosa), staphylococci, and viridans streptococci.

3

,

13 Because

mortality from untreated bacteremia caused by P. aeruginosa is so high,

65

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empiric regimens have traditionally included antimicrobials with antipseudomonal

activity.

Selecting an initial empiric regimen for a given patient should take into account the

patient’s risk of infection, likely pathogens, infection site-specific antibiotic efficacy,

and institutional susceptibility patterns. This point is especially true in areas

experiencing a growing frequency of multidrug-resistant organisms, such as MRSA,

VRE, and extended-spectrum β-lactamase (ESBL)-producing organisms (such as K.

pneumoniae and E. coli).

18 Patient-related considerations should include medical

stability, allergies, prior and concomitant antimicrobials, and organ dysfunction (e.g.,

renal or hepatic). Attempts should be made to identify low-risk patients for whom

oral antimicrobial therapy may be an option. Finally, dosing schedules, acquisition

costs, and the potential for significant toxicities should be considered. In addition to

broad-spectrum activity, antibacterial regimens should be bactericidal against the

infecting pathogen. However, no adequately controlled comparative trials of

bacteriostatic versus bactericidal antibiotics have been conducted in humans.

In summary, many organisms, including those recovered from surveillance

cultures, may be pathogens in B.C. Those associated with a high mortality rate during

the first 48 hours should be empirically treated pending culture and sensitivity

results. Therefore, an empiric regimen with optimal activity against commonly

isolated gram-negative bacilli (including P. aeruginosa) should be promptly

administered to B.C.

ORAL ANTIBIOTICS

Carefully selected (i.e., low risk) adult febrile neutropenic patients may be

candidates for oral antibiotic therapy, either as initial therapy or as follow-up to IV

antibiotics (sequential therapy).

2

,

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