Gillio A P, Faulkner L B, Alter B P, Reilly L, Klafter R, Heller G, Young D C, Lipton J M, Moore M A, O'Reilly R J
Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY 10021.
Stem Cells. 1993 Jul;11 Suppl 2:123-30. doi: 10.1002/stem.5530110820.
This report describes the response of 18 Diamond-Blackfan anemia (DBA) patients to recombinant human interleukin 3 (rhIL-3). rhIL-3 was administered s.c. once daily on an escalating dose schedule (0.5-10 micrograms/kg/day). The rhIL-3 dose was escalated every 21 days until erythroid response was attained, grade III or IV nonhematologic toxicity was observed, or the maximal rhIL-3 dose was reached. Four patients experienced clinically significant erythroid responses. Two of the responders were steroid-dependent and transfusion-independent, while two were steroid-independent and transfusion-dependent. Baseline clinical or laboratory parameters, in particular in vitro bone marrow erythroid progenitor assays, were not useful in predicting rhIL-3 response. Two of the responding patients remain on maintenance rhIL-3 without diminution of effect at 490 and 855+ days. rhIL-3 was discontinued in the other two responders because of the development of deep venous thrombi.
本报告描述了18例钻石-黑范贫血（DBA）患者对重组人白细胞介素3（rhIL-3）的反应。rhIL-3按剂量递增方案（0.5-10微克/千克/天）皮下注射，每日1次。每21天增加rhIL-3剂量，直至获得红系反应、观察到III级或IV级非血液学毒性或达到rhIL-3最大剂量。4例患者出现临床上显著的红系反应。其中2例反应者依赖类固醇且无需输血，另外2例不依赖类固醇但依赖输血。基线临床或实验室参数，尤其是体外骨髓红系祖细胞检测，对预测rhIL-3反应并无帮助。2例有反应的患者在490天和855天以上持续接受rhIL-3维持治疗，效果未减。另外2例反应者因发生深静脉血栓而停用rhIL-3。
