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Pregnancy is an absolute contraindication to RAI therapy. Previously, the use of

RAI was restricted to adults older than an arbitrary age of 20 to 35 years because it

was feared that RAI could result in genetic damage or neoplasia. However, its use in

adolescents is increasing after more than 50 years of clinical experience with RAI

showing that it is safe and effective.

184–187 There is no reported evidence of genetic

damage after

131

I ingestion, and the dose of radiation to the gonads is <3 rads, which

is comparable to other radiographic diagnostic tests (e.g., barium enemas).

188 The

incidence of leukemia or malignancy is no higher in recipients of

131

I than in

thyrotoxic patients treated with drugs or surgery.

185,189

In a retrospective review of 98

adolescents followed for 36 years after receiving

131

I, no cancers of the thyroid or

leukemia were reported.

187 One interesting finding is that patients receiving RAI

should be warned that they can set off radiation detectors at airport screening

terminals for up to 12 weeks after RAI and that they should carry documentation of

their treatment.

190,191

RAI is painless, effective, economical, and quick, but unsubstantiated fears about

radiation and malignancy, as well as the high incidence of hypothyroidism, may deter

its use. RAI could be used safely in this nonpregnant young patient. However, C.R.’s

prior use of iodides will dilute the

131

I pool. Thus, it will be impossible to achieve

therapeutic thyroid concentrations of RAI for as long as 3 to 6 months.

Treatment with Thioamides

PROPYLTHIOURACIL VERSUS METHIMAZOLE

CASE 52-15, QUESTION 4: C.R. is started on PTU 200 mg q8h after baseline FT4

and TSH levels have

been obtained. Three weeks later, she angrily complains that her symptoms are worse and that the medication

is not working; however, she reluctantly admits to missing doses because of difficulty swallowing, nausea,

vomiting, diarrhea, fatigue, a cough, and a sore throat. What are the advantages of using either PTU or

methimazole in the treatment of hyperthyroidism?

Both thioamides are effective in treating hyperthyroidism. The antithyroid

effectiveness of the thioamides primarily depends on their ability to block the

organification of iodines, thereby inhibiting thyroid hormone synthesis.

175,176 Thyroid

autoantibody synthesis may also be suppressed. In most hyperthyroid adults and

children, methimazole should be considered the thioamide of choice because of

increasing reports of hepatitis, some fatal, from PTU. PTU should be reserved for use

in thyroid storm, during the first trimester of pregnancy because of rare teratogenicity

from methimazole, and in those allergic to methimazole (except agranulocytosis and

hepatitis) who are not candidates for RAI or surgery.

30,169

Dosing and Administration

Methimazole is effective when administered initially as a single dose compared with

the multiple-dose regimen required with PTU to achieve a euthyroid state.

175,176

Although a single-dose regimen of PTU has been tried acutely, it is most effective

when given in divided doses. Compared to PTU, methimazole is also less

hepatototoxic, less expensive, requires daily ingestion of fewer numbers of tablets,

and is not associated with a bitter tablet taste. However, PTU is preferred in thyroid

storm because, unlike methimazole, it also blocks the peripheral conversion of T4

to

T3

.

192 Within 24 to 48 hours after PTU administration, a 25% to 40% reduction in

peripheral T3 production is seen, which contributes to PTU’s rapid onset. A

significantly greater fall in T3 concentration and the T3

:T4

ratio can be demonstrated

in hyperthyroid patients treated acutely with PTU and iodine than with methimazole

and iodides. Lastly, PTU is preferred over methimazole during the first trimester of

pregnancy (see Case 52-18).

CASE 52-15, QUESTION 5: Why was the thioamide therapy ineffective in C.R.? Was the dose of PTU

appropriate?

The inadequate response in C.R. suggests poor adherence to the thioamide dosing

regimen or a delayed response caused by prior iodide loading of the gland.

The onset of action of the thioamides is slow because they block the synthesis

rather than the release of thyroid hormone. Therefore, hormone secretion will

continue until the glandular stores of hormone are depleted. If adequate doses were

given, some improvement of clinical symptoms should be noted after 2 or 3 weeks.

176

The dosage of PTU is appropriate. Thioamide dosing consists of two phases:

initial therapy to achieve euthyroidism and maintenance therapy to achieve remission.

Initially, high blocking dosages of PTU (400–800 mg/day, depending on the severity

of the toxicosis) should be given in three or four divided doses, as in C.R.

164,175,176

Rarely, dosages of 1,200 mg/day of PTU or its equivalent may be required in patients

with severe disease or storm.

192 Equipotent doses of methimazole (which is 10 times

more potent than PTU on a mg-per-mg basis) can also be used. However, it is usually

unnecessary to use >40 mg/day of methimazole to restore a euthyroid state.

164,175,176

Toxicity is also less common (see Case 52-15, Questions 10 and 11). True resistance

to thioamides is rare; thus, most cases of unresponsiveness are caused by poor

patient adherence, as in C.R.

C.R.’s adherence is also hindered by the frequency of PTU administration. The

serum half-life of PTU is short (1.5 hours), but it is the intrathyroidal drug

concentrations that should determine the dosing intervals because they are most

clearly related to the drug’s antithyroid effects.

176 PTU must be dosed every 6 to 8

hours initially, or as frequently as every 4 hours in cases of severe hyperthyroidism

and thyroid storm. In contrast, methimazole has a serum half-life of 6 to 8 hours,

remains in the thyroid for 20 hours, and has a duration of activity of up to 40

hours.

176,193

p. 1060

p. 1061

Poor adherence is often difficult to ascertain and is more likely when multiple

daily doses are required. The best option for C.R. is to change to 30 to 40 mg of

methimazole, given once daily to improve adherence, or divided into two doses to

decrease GI distress. After methimazole is given for 4 to 6 weeks to achieve

euthyroidism, the daily dosage can be reduced gradually by 25% to 30% monthly to a

dosage that maintains euthyroidism, usually 5 to 10 mg/day of methimazole. If C.R.

remains hyperthyroid despite adequate doses of thioamides, then the most likely

reason is non-adherence.

Monitoring Therapy

CASE 52-15, QUESTION 6: What additional objective baseline data should be obtained to monitor both the

efficacy and toxicity of thioamides?

Before thioamides are administered, a baseline FT4 and TSH should be obtained.

A baseline white blood cell (WBC) count with differential can also help differentiate

the leukopenia associated with hyperthyroidism from drug-induced leukopenia and/or

agranulocytosis (see Case 52-15, Question 11). Baseline liver function tests can

assist in the evaluation of thioamide-induced hepatotoxicity (see Case 52-15,

Question 10). A repeat FT4 and TSH should be obtained after 4 to 6 weeks on

therapy and 4 to 6 weeks after any change in the dosing regimen. Once the patient is

euthyroid on maintenance dosages, thyroid function tests can be obtained every 3 to 6

months.

Duration of Therapy

CASE 52-15, QUESTION 7: How long should C.R. be continued on thioamide treatment?

Traditionally, thioamide therapy is continued for 1 to 2 years despite the lack of

data regarding the optimal treatment period.

164,175,176 The goal of treatment is to

control the symptoms of Graves’ disease until spontaneous remission occurs.

Graves’ disease remits spontaneously in about 30% to 50% of patients but relapse is

high.

194 Because it is unknown when or if remission will occur, it is understandable

why the optimal duration of therapy is unclear. Short-term therapy (i.e., <6 months)

has been advocated to save time and money and improve adherence because earlier

studies suggested remission rates comparable to a longer course of therapy.

However, short-term therapy is not recommended because longer follow-ups of

patients receiving short-term therapy have noted poorer remission rates.

175,176

Most data support a 12- to 18-month course of treatment to achieve remission rates

of approximately 60%.

175,176,195,196 Two prospective randomized trials found that

extending treatment from 6 to 18 months was beneficial but that 42 months of therapy

was not significantly better than 18 months.

195,196 However, one retrospective study of

patients treated for >12 months observed remission rates of only 17.5%.

197 These

conflicting results underscore the fact that determining the optimal treatment period is

confounded by the large variability that exists with regard to spontaneous remission.

Nevertheless, treatment periods of 1 to 2 years are justifiable in adherent patients.

Therapy can be reinstituted if hyperthyroidism reappears shortly after therapy is

discontinued. Methimazole can also be continued indefinitely if there are no side

effects, and treatment with either RAI or surgery is not desired. In C.R., this goal

might not be achievable, given her history of non-adherence.

PRECAUTIONS

CASE 52-15, QUESTION 8: Can thioamide therapy affect any of C.R.’s preexisting medical conditions?

Thyrotoxicosis can activate or intensify diabetes, primarily by increasing the basal

hepatic glucose production and the metabolism of insulin.

198 Therefore, effective

therapy with thioamides may restore control of C.R.’s type 2 diabetes.

C.R.’s arthritis should not be affected, although thioamides have been associated

with the development of lupus erythematosus (LE), lupus-like syndromes, and

vasculitis.

176,199 These adverse drug reactions are rare; the incidence is <0.1%.

Lupus-like syndromes include skin ulcers, splenomegaly, migratory polyarthritis,

pleuritis and pericarditis, periarteritis, and renal abnormalities. Serologic

abnormalities may also occur with these connective tissue disorders and include

hyperglobulinemia, positive LE preparations, and positive antinuclear antibodies.

Recovery occurs with adequate steroid therapy and withdrawal of the thioamides.

Because cross-reaction between methimazole and PTU is likely to occur, patients

exhibiting these reactions should be treated with surgery or RAI. C.R.’s treatment

should be monitored with this lupus-like adverse effect in mind, but the occurrence of

this syndrome is so uncommon that a trouble-free course of therapy can be

anticipated.

ADJUNCTIVE THERAPY

CASE 52-15, QUESTION 9: What adjunctive therapy might help alleviate some of C.R.’s symptoms while

awaiting the onset of thioamide’s effects?

Iodides (see Case 52-15, Question 2) , β-adrenergic blocking agents without

intrinsic sympathomimetic activity, or calcium channel blockers can be used acutely

to ameliorate some of C.R.’s symptoms.

200,201 Because iodides were previously

ineffective in C.R., a β-blocker should be tried.

β-Adrenergic blocking agents rapidly decrease the nervousness, palpitations,

fatigue, weight loss, diaphoresis, heat intolerance, and tremor associated with

thyrotoxicosis, probably because many of the signs and symptoms mimic sympathetic

overactivity.

164,200 An increase in the number of β-adrenergic receptors rather than an

elevation in catecholamine levels is probably responsible for this overactivity.

Because the underlying disease process and thyroid hormone levels are not affected

significantly by β-blockers, patients generally remain mildly symptomatic and fail to

gain weight. For this reason, they should not be used as the sole treatment for

thyrotoxicosis.

All β-blockers without intrinsic sympathetic activity (e.g., atenolol, metoprolol,

propranolol) are effective in alleviating the hyperthyroid symptoms, but propranolol

is the only β-blocker that significantly inhibits peripheral conversion of T4

to T3

.

200

Thyroid function tests are generally not affected except for a mild decrease in the T3

level.

In summary, β-blockers are (a) effective adjuncts in the management of thyroid

storm, (b) useful to prepare patients for surgery, and (c) useful in the short-term

management of thyrotoxicosis during pregnancy.

180,192,200 Surprisingly, propranolol

also improves many of the neuromuscular manifestations of hyperthyroidism,

including thyrotoxic periodic paralysis. Current guidelines recommend elderly

patients, those with concomitant heart disease, and those with a resting heart rate

over 90 beats/minute receive adjunctive therapy with β-blockers.

34

Diltiazem or verapamil are effective alternatives when β-blockers are

contraindicated.

201 Diltiazem 120 mg TID or QID can be tried. The dihydropyridine

calcium channel blockers are unlikely to be effective.

Because of C.R.’s history of diabetes, the effects of β-adrenergic blocking drugs

on patients with diabetes must be considered (see Chapter 53, Diabetes Mellitus). If

β-blockers are instituted, a cardioselective β-blocker would be a better choice for

patients

p. 1061

p. 1062

on therapy that can cause hypoglycemia. The appropriate dosage should be based

on clinical and objective improvement of hyperthyroid symptoms, such as a reduction

in heart rate. Metoprolol 25 to 50 mg BID can be started initially and the dosage

titrated to maintain the heart rate at <90 beats/minute. Otherwise, diltiazem,

verapamil, or a retrial of iodides is warranted.

ADVERSE EFFECTS

CASE 52-15, QUESTION 10: A pruritic area over the pretibial aspects of both legs as well as several

maculopapular erythematous patches and abdominal tenderness were noticed during C.R.’s physical

examination. Do these reactions require the discontinuation of her PTU?

Thioamide Rash

Although C.R. may be experiencing a drug rash from PTU, pretibial myxedema or the

dermopathy of Graves’ disease may also be possibilities because of the location of

the pruritic area. About 4% of patients with Graves’ disease who exhibit infiltrative

exophthalmos also have dermatologic changes. The skin is thickened, erythematous,

and non-pitting because of mucopolysaccharide infiltration and accentuation of hair

follicles. Pruritus or pain may be present. Treatment includes topical corticosteroids,

control of the Graves’ disease, and reassurance.

Thioamides can produce a maculopapular pruritic rash in 5% to 6% of treated

patients.

176 The rash can occur at any time but is more common early in therapy. If the

rash is mild, drug therapy can be continued while the patient’s symptoms are treated

with an antihistamine and a topical steroid; such rashes generally subside

spontaneously. Alternatively, another thioamide can be substituted because crosssensitivity to this side effect is uncommon. Thioamides should be stopped if the rash

is urticarial in nature or associated with systemic manifestations (e.g., fever,

arthralgias).

Hepatitis

C.R.’s symptoms of nausea, vomiting, diarrhea, fatigue, and abdominal tenderness

require further evaluation. Her symptoms could be consistent with mild GI side

effects from her PTU therapy, impending thyroid storm from non-adherence (see Case

52-22, Question 1) or more seriously, PTU-induced hepatitis. PTU has been

associated with 0.1% severe hepatotoxicity, leading to liver transplants and fatalities

primarily in children.

30,169,202,203 Although usually hepatocellular in nature, cholestasis,

hepatic necrosis, and fulminant hepatic failures have been reported.

168,170,176 Transient

elevations in transaminases occur in approximately 30% of asymptomatic patients

within the first 2 months of PTU therapy and may not require drug discontinuation.

168

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