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Thalidomide use in pediatric patients.

Olga Bessmertny¹, Trinh Pham

¹Pediatric/Hematology/Oncology/Bone Marrow Transplantation, Columbia Presbyterian Medical Center, New York, NY, USA.

The Annals of Pharmacotherapy

|March 16, 2002

Summary

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Thalidomide shows effectiveness in chronic graft-versus-host disease (GVHD) and aphthous ulcers in HIV-infected children. Its use is recommended as a last resort for pediatric patients when other treatments fail.

Area of Science:

Pediatric pharmacology
Immunology
Dermatology

Background:

Thalidomide, a teratogenic drug, has demonstrated immunomodulatory properties.
Its use in pediatric populations requires careful consideration due to potential side effects.

Purpose of the Study:

To review the existing literature on thalidomide's efficacy and safety in pediatric patients.
To identify specific conditions where thalidomide may be beneficial in children.

Main Methods:

A comprehensive literature search was conducted using MEDLINE (1966-February 2001).
Keywords included thalidomide, child, graft-versus-host disease, cancer, HIV, Crohn's disease, Behçet's disease, and lupus erythematosus.
References from identified articles were also reviewed.

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Main Results:

Thalidomide appears effective for chronic graft-versus-host disease (GVHD) and aphthous ulcers in HIV-infected children.
Limited evidence suggests efficacy for cutaneous manifestations of Behçet's disease, Crohn's disease, and lupus in children, with high recurrence rates upon discontinuation.
Efficacy in acute GVHD and cancer was not clearly established.

Conclusions:

Thalidomide should be considered a last-resort treatment option for pediatric patients.
Preferential use in male or prepubescent female patients is suggested.
Careful risk-benefit assessment is crucial due to potential teratogenicity and disease recurrence.