The pathophysiology of pure red cell aplasia: Implications for therapy
The pathophysiology of pure red cell aplasia: Implications for therapy
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DOI：
10.1182/blood.v87.11.4831.bloodjournal87114831
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发表时间：
1996-06-01
期刊：
影响因子：
20.3
通讯作者：
Abkowitz, JL
中科院分区：
文献类型：
--
作者：
Charles, RJ;Sabo, KM;Abkowitz, JL
为了确定骨髓培养在确定纯红细胞发育不全的自然历史和治疗反应中的作用，我们对37例患者进行了研究。患者在特定治疗前（n = 21）或治疗失败时（n = 16）在华盛顿大学进行评估。评估包括医疗和药物暴露史、体格检查、胸部x线或计算机断层扫描以排除胸腺瘤、淋巴细胞免疫表型研究、抗核抗体和类风湿因子测定、骨髓细胞遗传学和骨髓祖细胞培养。对保存血清的27例患者进行回顾性Southern分析，检测人细小病毒B19。临床随访记录治疗效果。培养中正常的红细胞爆发形成单位（BFU-E）生长（bbb30次爆发/10(5)个骨髓单核细胞[MMNC]）被证明是临床反应的一个杰出预测指标，29例红细胞爆发频率正常的个体中有27例对免疫调节治疗有反应（敏感性96%，特异性78%，预测值93%，双尾卡方分析P = 0.0001）。总的来说，28名患者对免疫调节疗法或停药有反应。24例患者获得正常的血细胞比容（完全缓解[CR]），另外4例患者成为不依赖输血（部分缓解）。虽然应答的患者通常需要几种治疗，但在最后一次随访（中位5年）时，24例获得CR的患者中有20例（83%）在没有维持治疗的情况下维持了正常的红细胞压积。相比之下，8例体外BFU-E生长不良的患者(
To determine the utility of marrow culture in defining the natural history and therapeutic response of pure red cell aplasia we have studied 37 patients. Patients were evaluated at the University of Washington before specific therapies (n = 21) or at the time of treatment failure (n = 16). Evaluation included a medical and drug exposure history, a physical examination, a chest x-ray or computed tomography to rule out thymoma, lymphocyte immunophenotype studies, anti-nuclear antibody and rheumatoid factor determinations, marrow cytogenetics, and marrow progenitor cell cultures. Retrospective Southern analyses to detect human parvovirus B19 was performed in the 27 patients for whom sera was stored. Clinical follow-up was obtained to document therapeutic responses. Normal burst forming unit-erythroid (BFU-E) growth (>30 bursts/10(5) marrow mononuclear cells [MMNC]) in culture proved an outstanding predictor of clinical response, as 27 of 29 individuals with normal frequencies of erythroid bursts in culture responded to immunomodulating therapies (sensitivity 96%, specificity 78%, predictive value 93%, P = .0001 with two-tailed chi square analysis). Overall, 28 patients responded to either immunomodulating therapies or drug withdrawal. Twenty-four patients obtained a normal hematocrit (complete response [CR]) and 4 additional patients became transfusion independent (partial response). Although responding patients often required several therapies, 20 of 24 (83%) patients who obtained a CR have sustained a normal hematocrit without maintenance therapy at the time of last follow-up (median 5 years). In contrast, of 8 patients with poor in vitro BFU-E growth (