Department of Hematology, Beijing Friendship Hospital, Capital Medical University, Beijing, China.
Department of General Practice, Beijing Friendship Hospital, Capital Medical University, Beijing, China.
Hematol Oncol. 2022 Aug;40(3):390-399. doi: 10.1002/hon.3014. Epub 2022 May 15.
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative approach for primary hemophagocytic lymphohistiocytosis (pHLH), but data on adult patients are scarce. Here we present an 8-year experience on HSCT for adult pHLH to reveal the benefits and risks in this population. A total of 29 adult pHLH patients entered this study, at a median follow-up of 29 months (3-112 months), the 5-year probability of survival was 60%. Six patients rejected HSCT, of whom 1 alive in complete response (CR). In 23 patients who underwent HSCT, 5-year survival post-HSCT overall was 73%, and in haploidentical HSCT (haplo-HSCT) cases, 71%. Patients who achieved CR at HSCT had a better outcome than those of partial response (92% vs. 47%, p = 0.013). Neither the use of HLA mismatched donor (75% vs. 72%, p = 0.996) nor the use of donor with monoallelic mutation (74% vs. 71%, p = 0.901) affected the prognosis. Hemophagocytic lymphohistiocytosis status of CR at HSCT was a positive prognostic factor. We concluded that HSCT is a promising method to cure adult-onset pHLH. Achieving CR before HSCT contributes to better outcome. Haplo-HSCT is safe and effective for adult pHLH patients, donors with monoallelic mutations in pHLH related genes but normal cytotoxic functions are reliable.
异基因造血干细胞移植（HSCT）是原发性噬血细胞性淋巴组织细胞增多症（pHLH）的唯一根治方法，但成人患者的数据很少。在此，我们报告了 8 年成人 pHLH 患者 HSCT 的经验，以揭示该人群的获益和风险。共有 29 例成人 pHLH 患者入组本研究，中位随访时间为 29 个月（3-112 个月），5 年生存率为 60%。6 例患者拒绝 HSCT，其中 1 例在完全缓解（CR）时存活。在 23 例接受 HSCT 的患者中，HSCT 后 5 年总生存率为 73%，在单倍体相合 HSCT（haplo-HSCT）病例中为 71%。HSCT 时达到 CR 的患者比部分缓解的患者预后更好（92% vs. 47%，p=0.013）。HLA 错配供者的使用（75% vs. 72%，p=0.996）或单等位基因突变供者的使用（74% vs. 71%，p=0.901）均不影响预后。HSCT 时噬血细胞性淋巴组织细胞增多症的 CR 状态是一个阳性预后因素。我们得出结论，HSCT 是治疗成人发病的 pHLH 的一种有前途的方法。HSCT 前达到 CR 有助于获得更好的结局。haplo-HSCT 是安全有效的，对于 pHLH 相关基因单等位基因突变但正常细胞毒性功能的供者是可靠的。
