Conter V, Reciputo A, Arrigo C, Bozzato N, Sala A, Aricò M
Department of Pediatrics, University of Milan, Italy.
Haematologica. 1996 Sep-Oct;81(5):468-71.
Langerhans' cell histiocytosis (LCH) is an uncommon disorder of childhood, formerly referred to histiocytosis X. A significant proportion of children with disseminated disease may undergo progression to a fatal outcome despite chemotherapy with single or multiple agents. Only six cases of LCH treated with BMT have been reported in the literature, including two cases of autologous BMT. Of them, only one was less than 14 years of age. We describe a 4-year-old child whose disseminated, refractory Langerhans' cell histiocytosis was not controlled by front-line monotherapy with etoposide, nor by rescue treatment with combined chemotherapy (vinblastine and etoposide) and immunotherapy (steroids and cyclosporine). Due to the high risk of fatal progressive disease, he underwent bone marrow transplantation from his HLA-identical sister who was heterozigous for beta-thalassemia. On day 24 after transplantation marrow reconstitution was evident, with WBC count 2.3 x 10(9)/L, neutrophil count > 0.5 x 10(9)/L, and platelet count 72 x 10(9)/L. Engraftment was demonstrated by PCR DNA analysis. The patient was discharged on day 25. After transplantation he experienced fever for 11 days and developed signs of grade I cutaneous and intestinal graft-versus-host disease, that was treated with methylprednisolone from days 11 to day 68 (1 mg/kg/day for 18 days, then tapered). He became transfusion independent on day 24; the hemoglobin value was 7.5 g/dL on day 54 and has remained > 10 g/dL since day 200. Features of heterozygous beta-thalassemia have been evident since then. Bone marrow aspirate was normal on days 25 and 94. At the time of this writing he remains in excellent condition, disease and treatment free, 25 months after transplantation. Although limited, current experience suggests that bone marrow transplantation has the potential to cure refractory Langerhans' cell histiocytosis.
朗格汉斯细胞组织细胞增多症（LCH）是一种儿童期罕见疾病，以前称为组织细胞增多症X。尽管使用单药或多药化疗，相当一部分患有播散性疾病的儿童仍可能进展至致命结局。文献中仅报道了6例接受骨髓移植治疗的LCH病例，其中包括2例自体骨髓移植。其中，只有1例年龄小于14岁。我们描述了一名4岁儿童，其播散性、难治性朗格汉斯细胞组织细胞增多症既未被依托泊苷一线单药治疗控制，也未被联合化疗（长春花碱和依托泊苷）及免疫治疗（类固醇和环孢素）的挽救治疗控制。由于致命性进展性疾病风险高，他接受了来自其 HLA 相同的、β地中海贫血杂合子的姐姐的骨髓移植。移植后第24天骨髓重建明显，白细胞计数为2.3×10⁹/L，中性粒细胞计数>0.5×10⁹/L，血小板计数为72×10⁹/L。通过PCR DNA分析证实了植入。患者于第25天出院。移植后他发热11天，并出现了I级皮肤和肠道移植物抗宿主病的体征，从第11天至第68天用甲泼尼龙治疗（18天内每日1mg/kg，然后逐渐减量）。他在第24天不再依赖输血；第54天血红蛋白值为7.5g/dL，自第200天起一直>10g/dL。从那时起，杂合子β地中海贫血的特征就很明显。移植后第25天和第94天骨髓穿刺正常。在撰写本文时，他状况良好，移植后25个月无疾病且无需治疗。尽管经验有限，但目前的经验表明骨髓移植有可能治愈难治性朗格汉斯细胞组织细胞增多症。
