Brodsky R A, Petri M, Smith B D, Seifter E J, Spivak J L, Styler M, Dang C V, Brodsky I, Jones R J
Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, USA.
Ann Intern Med. 1998 Dec 15;129(12):1031-5. doi: 10.7326/0003-4819-129-12-199812150-00007.
Immunoablative high-dose cyclophosphamide without stem-cell rescue induces durable, complete remission in most patients with aplastic anemia.
To determine the efficacy of high-dose cyclophosphamide in various refractory, severe autoimmune diseases.
Prospective phase II study.
Johns Hopkins University (Baltimore, Maryland) and Hahnemann University (Philadelphia, Pennsylvania).
Eight patients with refractory, severe autoimmune disease.
Immunoablative high-dose cyclophosphamide (50 mg/kg of body weight per day) for 4 consecutive days.
Clinical and laboratory variables of autoimmune disease.
Seven patients improved markedly: Five achieved complete remission and two achieved partial remission. Four patients have remained in continuous complete remission for 3 to 21 months, and two patients in partial remission continue to improve after 14 and 19 months of follow-up. High-dose cyclophosphamide was well tolerated; median times to a neutrophil count of 0.5 x 10(9) cells/L and platelet transfusion independence were 17 and 16 days, respectively.
Immunoablative high-dose cyclophosphamide without stem-cell rescue can induce complete remission in patients with refractory, severe autoimmune disease. Reemergence of marrow function is similar to that seen after autologous transplantation and does not carry the risk for reinfusion of autoaggressive lymphocytes with the autograft.
无干细胞救援的免疫清除性高剂量环磷酰胺可使大多数再生障碍性贫血患者获得持久的完全缓解。
确定高剂量环磷酰胺在各种难治性重症自身免疫性疾病中的疗效。
前瞻性II期研究。
约翰霍普金斯大学（马里兰州巴尔的摩）和哈内曼大学（宾夕法尼亚州费城）。
8例难治性重症自身免疫性疾病患者。
免疫清除性高剂量环磷酰胺（每日50mg/kg体重），连续4天。
自身免疫性疾病的临床和实验室变量。
7例患者明显改善：5例获得完全缓解，2例获得部分缓解。4例患者持续完全缓解3至21个月，2例部分缓解患者在随访14个月和19个月后仍持续改善。高剂量环磷酰胺耐受性良好；中性粒细胞计数达到0.5×10⁹/L和不再需要血小板输注的中位时间分别为17天和16天。
无干细胞救援的免疫清除性高剂量环磷酰胺可使难治性重症自身免疫性疾病患者获得完全缓解。骨髓功能的恢复与自体移植后相似，且不存在自体移植中自身攻击性淋巴细胞回输的风险。
