Abkowitz J L, Powell J S, Nakamura J M, Kadin M E, Adamson J W
Am J Hematol. 1986 Dec;23(4):363-71. doi: 10.1002/ajh.2830230408.
Pure red cell aplasia (PRCA) results from the failure of erythrocyte differentiation and may respond to immunosuppressive therapies. We have treated nine patients with PRCA refractory to steroids and/or cyclophosphamide with anti-thymocyte globulin (ATG). Six patients had normal numbers of erythroid bursts (from erythroid burst-forming units) or erythroid colonies (from erythroid colony-forming units) detectable in vitro, and all responded to therapy with ATG. In vitro studies suggested T-cell inhibition of erythropoiesis in four of these six patients and humorally mediated erythroid suppression in one. In three individuals, virtually no erythroid progenitors were detected in marrow culture. None of these patients responded to ATG. Myelofibrosis, 5q- chromosomal abnormality, or the subsequent development of thrombocytopenia in these individuals suggested that PRCA resulted from an intrinsic stem cell disorder. Our studies demonstrate that ATG is effective therapy for PRCA, and it may be especially useful in children or other patients in whom alkylating agents are not appropriate. We also confirm that erythroid growth in marrow culture predicts those patients who will respond to ATG or other immunosuppressive therapies.
纯红细胞再生障碍性贫血（PRCA）是由红细胞分化失败引起的，可能对免疫抑制疗法有反应。我们用抗胸腺细胞球蛋白（ATG）治疗了9例对类固醇和/或环磷酰胺难治的PRCA患者。6例患者在体外可检测到正常数量的红系爆式集落（来自红系爆式集落形成单位）或红系集落（来自红系集落形成单位），且所有患者对ATG治疗均有反应。体外研究表明，这6例患者中有4例存在T细胞对红细胞生成的抑制，1例存在体液介导的红系抑制。在3例个体中，骨髓培养几乎未检测到红系祖细胞。这些患者均对ATG无反应。这些个体出现骨髓纤维化、5号染色体长臂缺失或随后发生血小板减少，提示PRCA是由内在干细胞疾病引起的。我们的研究表明，ATG是治疗PRCA的有效疗法，对儿童或其他不适合使用烷化剂的患者可能特别有用。我们还证实，骨髓培养中的红系生长可预测哪些患者会对ATG或其他免疫抑制疗法有反应。
