Hamidieh Amir Ali, Pourpak Zahra, Hashemi Susan, Yari Kolsoum, Fazlollahi Mohammad Reza, Movahedi Masoud, Behfar Maryam, Moin Mostafa, Ghavamzadeh Ardeshir
Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
Eur J Haematol. 2014 Apr;92(4):331-6. doi: 10.1111/ejh.12244. Epub 2014 Jan 10.
Primary hemophagocytic lymphohistiocytosis (HLH) is a life-threatening condition that clinically characterized by fever, hepatosplenomegaly, and cytopenia. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment option for patients diagnosed with primary HLH.
In this prospective study, we analyzed the outcome of 10 pediatric patients with primary HLH who had received HSCT, using reduced-intensity conditioning (RIC) regimen from 2007 to 2012. The median age at transplantation was 22.6 months (range: 6-60). All of the patients received the same RIC regimen based on the use of fludarabine in combination with melphalan and horse antithymocyte globulin (ATG). Cyclosporine and methylprednisolone were used as graft-vs.-host disease (GvHD) prophylaxis.
Hematopoietic engraftment occurred in all patients. At the present time, 8 patients with a median follow-up of 39 months are still alive and all of them are disease free. Acute and chronic GvHD developed in 6 and 2 patients, retrospectively. Two patients died of sepsis and chronic GvHD during the study.
Because of pretransplant infections caused by underlying immunodeficiency in patients with primary HLH, the use of less toxic regimen with RIC seems to be highly effective in this regard. Recipients of RIC transplant, with either full or mixed chimerism, had a long-term survival rate with no manifestation of primary HLH symptoms.
原发性噬血细胞性淋巴组织细胞增生症（HLH）是一种危及生命的疾病，临床特征为发热、肝脾肿大和血细胞减少。造血干细胞移植（HSCT）是诊断为原发性HLH患者的唯一治愈性治疗选择。
在这项前瞻性研究中，我们分析了2007年至2012年期间10例接受HSCT的原发性HLH儿科患者的结局，采用了减低强度预处理（RIC）方案。移植时的中位年龄为22.6个月（范围：6 - 60个月）。所有患者均接受基于氟达拉滨联合美法仑和马抗胸腺细胞球蛋白（ATG）的相同RIC方案。环孢素和甲泼尼龙用于预防移植物抗宿主病（GvHD）。
所有患者均实现造血植入。目前，8例患者中位随访39个月，仍存活且均无疾病。回顾性分析显示，6例和2例患者分别发生了急性和慢性GvHD。研究期间，2例患者死于败血症和慢性GvHD。
由于原发性HLH患者潜在免疫缺陷导致移植前感染，使用毒性较小的RIC方案在这方面似乎非常有效。接受RIC移植的患者，无论是完全嵌合还是混合嵌合，均有长期生存率，且无原发性HLH症状表现。
