Matloub Y H, Bostrom B, Golembe B, Priest J, Ramsay N K
Division of Pediatric Hematology-Oncology, Children's Hospital of Western Ontario, London, Canada.
Am J Pediatr Hematol Oncol. 1994 May;16(2):104-6.
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.
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).
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.
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.
该治疗试验的目的是尝试使用最成功且副作用最小的现有药物，优化对缺乏合适骨髓供体的儿童重度和中度再生障碍性贫血的治疗。
四个机构开展了一项针对儿童重度再生障碍性贫血的试点研究。治疗方案包括抗胸腺细胞球蛋白（ATG）、泼尼松和环孢素A。共纳入12例患者，其中11例可进行评估。所有患者均患有重度再生障碍性贫血（SAA）；3例患有肝炎诱发的重度再生障碍性贫血（HI-SAA）。
在11例可评估的患者中，8例血液计数恢复正常，获得缓解。3例HI-SAA患者中有2例对治疗有反应。
我们试点研究的结果优于以往的治疗试验。所有有反应的患者均实现了完全缓解，即血液计数恢复到正常范围内。
