Monitoring

Patients should be reviewed regularly while they are receiving hormonal treatment. They should be closely monitored to assess response to treatment and developmental progress. It is important to give parents adequate information about the long-term prognosis, the adverse effects of treatments, and the underlying etiology. They should be advised to seek urgent medical attention if the child becomes ill.

Hormonal therapy monitoring

In view of the adverse effects from hormonal therapy (including hypertension, hyperglycemia, increased susceptibility to infections, increased appetite, weight gain, irritability, gastric irritation, or altered sleep-wake pattern), clinical assessment, including measurement of blood pressure and urinalysis for glycosuria, should be performed both before and regularly during the course of hormonal treatment. The adverse effects of long-term corticosteroid therapy may be avoided by limiting the length of treatment.

Adrenocorticotropic hormone (ACTH) intramuscular injection should be given by a healthcare professional who can deal with allergic reactions and in a hospital setting. Necessary equipment and drugs to deal with anaphylaxis should be kept ready. Reactions such as marked redness, pain at reaction site, urticaria, pruritus, flushing, and dyspnea should be looked for. If any reactions are noted, the treatment should be switched to prednisone.

Vigabatrin monitoring

Vigabatrin-induced peripheral visual field defects are well recognized and irreversible. Visual fields should be assessed before treatment with vigabatrin, but this should not delay initiation of treatment. No consensus exists with regard to the most appropriate monitoring schedule. Vigabatrin-associated visual field defects have been reported to occur within 6 weeks of commencing therapy.[58] Regular surveillance is required, but this is difficult in the pediatric population. Where possible, visual field testing at baseline, 3 months, and every 6 months while on treatment is recommended.

While there is some evidence that vigabatrin may precipitate a movement disorder, a direct causal relationship is not clear. Of those who develop a movement disorder, in 25% it resolves when therapy is discontinued; in 50% it persists despite withdrawing vigabatrin; and in the remainder it resolves spontaneously without modifying the vigabatrin dose. There is currently insufficient evidence to postulate whether this movement disorder is reflective of the underlying etiology or if vigabatrin therapy is potentially causative.[61]

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