Antithymocyte globulin, cyclosporine, and prednisone for the treatment of severe aplastic anemia in children. A pilot study.

The American journal of pediatric hematology/oncology Pub Date : 1994-05-01

Y H Matloub, B Bostrom, B Golembe, J Priest, N K Ramsay

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Abstract

Purpose: The aim of the therapeutic trial was to try to optimize the treatment of severe and moderate aplastic anemia in children who lack a suitable bone marrow donor using the most successful available drugs, with the least amount of side effects.

Patients and methods: A pilot study for the treatment of severe aplastic anemia in children was conducted by four institutions. The treatment protocol included antithymocyte globulin (ATG), prednisone, and cyclosporine A. Twelve patients were enrolled, and 11 were evaluable. All patients had severe aplastic anemia (SAA); three had hepatitis-induced severe aplastic anemia (HI-SAA).

Results: Of 11 evaluable patients, eight have responded with normalization of their blood counts. Two of the three patients with HI-SAA responded to the therapy.

Conclusion: The results of our pilot study compare favorably with previous therapeutic trials. All the patients who responded achieved complete response, i.e., restoration of blood counts to within the normal range.

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抗胸腺细胞球蛋白、环孢素和强的松治疗儿童严重再生障碍性贫血。一项初步研究。

目的:治疗性试验的目的是尝试使用最成功的现有药物，以最小的副作用，优化治疗缺乏合适骨髓供体的重度和中度再生障碍性贫血儿童。患者和方法:四家机构对儿童重度再生障碍性贫血的治疗进行了一项试点研究。治疗方案包括抗胸腺细胞球蛋白(ATG)、强的松和环孢素a。纳入12例患者，其中11例可评估。所有患者均为重度再生障碍性贫血(SAA);3例为肝炎所致严重再生障碍性贫血(HI-SAA)。结果:在11例可评估的患者中，8例患者的血细胞计数恢复正常。三名HI-SAA患者中有两名对治疗有反应。结论:我们的初步研究结果与以前的治疗试验比较有利。所有有反应的患者均达到完全缓解，即血细胞计数恢复到正常范围内。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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The American journal of pediatric hematology/oncology

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