Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah Medical Center, Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.
Immunology Division, The Garvan Institute of Medical Research Graduate Research School, University of New South Wales, Sydney, NSW, Australia.
Front Immunol. 2022 May 27;13:879994. doi: 10.3389/fimmu.2022.879994. eCollection 2022.
Autoimmune cytopenia (AIC) is a rare complication post hematopoietic stem cell transplantation (HSCT), with a higher incidence in nonmalignant diseases. The etiology of post-HSCT AIC is poorly understood, and in many cases, the cytopenia is prolonged and refractory to treatment. Diagnosis of post-HSCT AIC may be challenging, and there is no consensus for a standard of care. In this retrospective study, we summarize our experience over the past five years with post-HSCT AIC in pediatric patients with osteopetrosis and other nonmalignant diseases. All pediatric patients who underwent HSCT for nonmalignant diseases at Hadassah Medical Center over the past five years were screened for post-HSCT AIC, and data were collected from the patient's medical records. From January 2017 through December 2021, 140 pediatric patients underwent HSCT for osteopetrosis (n=40), and a variety of other nonmalignant diseases. Thirteen patients (9.3%) presented with post-HSCT AIC. Of these, 7 had osteopetrosis (17.5%), and 6 had other underlying nonmalignant diseases. Factors associated with developing AIC included unrelated or non-sibling family donors (n=10), mixed chimerism (n=6), and chronic GvHD (n=5). Treatment modalities included steroids, IVIG, rituximab, bortezomib, daratumumab, eltrombopag, plasmapheresis, and repeated HSCT. Response to treatment was variable; Seven patients (54%) recovered completely, and three patients (23%) recovered partially, still suffering from mild-moderate thrombocytopenia. Three patients died (23%), two following progressive lung disease and one from sepsis and multi-organ failure after a 3 HSCT. In our experience, post-HSCT AICs in pediatric patients with nonmalignant diseases may pose a challenging post-transplant complication with a variable presentation and a wide spectrum of severity. A relatively high prevalence is seen in patients with osteopetrosis, possibly due to difficult engraftment and high rates of mixed chimerism. There is a dire need for novel treatment modalities for better management of the more severe and refractory cases.
自身免疫性血细胞减少症（AIC）是造血干细胞移植（HSCT）后的一种罕见并发症，在非恶性疾病中发病率较高。HSCT 后 AIC 的病因尚不清楚，在许多情况下，血细胞减少症持续时间长且对治疗有抗性。HSCT 后 AIC 的诊断可能具有挑战性，并且没有共识的治疗标准。在这项回顾性研究中，我们总结了过去五年中我们在患有成骨不全症和其他非恶性疾病的儿科患者中治疗 HSCT 后 AIC 的经验。过去五年中，在哈达萨医疗中心接受非恶性疾病 HSCT 的所有儿科患者均接受了 HSCT 后 AIC 的筛查，并从患者的病历中收集了数据。2017 年 1 月至 2021 年 12 月，140 名儿科患者因成骨不全症（n=40）和各种其他非恶性疾病接受了 HSCT。13 名患者（9.3%）出现了 HSCT 后 AIC。其中，7 例为成骨不全症（17.5%），6 例为其他潜在非恶性疾病。发生 AIC 的相关因素包括无关或非亲缘供体（n=10）、混合嵌合体（n=6）和慢性 GvHD（n=5）。治疗方式包括类固醇、IVIG、利妥昔单抗、硼替佐米、达雷妥尤单抗、艾曲波帕、血浆置换和重复 HSCT。治疗反应各不相同；7 名患者（54%）完全缓解，3 名患者（23%）部分缓解，仍患有轻中度血小板减少症。3 名患者死亡（23%），2 例死于进行性肺病，1 例死于 3 次 HSCT 后脓毒症和多器官衰竭。根据我们的经验，患有非恶性疾病的儿科患者的 HSCT 后 AIC 可能是一种具有挑战性的移植后并发症，其表现和严重程度差异很大。在成骨不全症患者中，这种情况较为常见，可能是由于植入困难和混合嵌合体率较高所致。需要新的治疗方法来更好地管理更严重和难治性病例。
