Hess Jennifer, Su Leon, Nizzi Frank, Beebe Kristen, Magee Kyrie, Salzberg Dana, Stahlecker Jennifer, Miller Holly K, Adams Roberta H, Ngwube Alexander
Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, Arizona.
Mayo Clinic, Scottsdale, Arizona.
Transfusion. 2018 Sep;58(9):2122-2127. doi: 10.1111/trf.14907. Epub 2018 Sep 4.
After hematopoietic stem cell transplantation (HSCT) autoimmune hemolytic anemia (AIHA) is a known and fairly common complication. It is often refractory to conventional therapies including corticosteroids, intravenous immunoglobulin, splenectomy, and the more recently described use of monoclonal antibodies. The high morbidity associated with these severe persistent cases elucidates the gaps in alternative therapies available for treatment.
We described the successful use of abatacept for severe refractory AIHA after HSCT in three patients.
Three pediatric patients with refractory AIHA after allogeneic stem cell transplantation were observed to be unresponsive to multitude immunosuppressive therapies, resulting in persistent transfusion dependency. Treatment with abatacept, a fusion protein that inhibits T-cell activation by binding to CD80/CD86 on antigen-presenting cells (APCs), thus blocking the required CD28 interaction between APCs and T cells, resulted in the resolution of hemolysis.
Abatacept may provide significant clinical benefit in the management of AIHA after HSCT.
造血干细胞移植（HSCT）后，自身免疫性溶血性贫血（AIHA）是一种已知且较为常见的并发症。它通常对包括皮质类固醇、静脉注射免疫球蛋白、脾切除术以及最近描述的单克隆抗体使用在内的传统疗法具有耐药性。这些严重持续性病例所伴随的高发病率揭示了可供治疗的替代疗法存在的差距。
我们描述了阿巴西普在3例HSCT后严重难治性AIHA患者中的成功应用。
观察到3例异基因干细胞移植后难治性AIHA的儿科患者对多种免疫抑制疗法无反应，导致持续依赖输血。阿巴西普是一种融合蛋白，通过与抗原呈递细胞（APC）上的CD80/CD86结合来抑制T细胞活化，从而阻断APC与T细胞之间所需的CD28相互作用，用其治疗可使溶血得到缓解。
阿巴西普在HSCT后AIHA的管理中可能提供显著的临床益处。
