Comparable long-term outcomes between upfront haploidentical and identical sibling donor transplant in aplastic anemia: a national registry-based study

Abstract

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains a curative option for severe aplastic anemia (SAA), and transplantation from identical sibling donors (ISDs) has been recommended as a first-line treatment. Haploidentical donor (HID) transplantation for SAA has made great advances; thus, an increased role of HID-SCT in SAA should be considered. We performed a national registry-based analysis comparing long-term outcomes in the upfront HID or upfront ISD SCT setting. A total of 342 SAA patients were enrolled, with 183 patients receiving HID SCT and 159 receiving ISD SCT. The estimated 9-year overall survival (OS) and failure-free survival (FFS) were 87.1±2.5% and 89.3±3.7% (P = 0.173) and 86.5±2.6% vs. 88.1±3.8% (P = 0.257) for patients in the HID and ISD SCT groups, respectively. Transplantation from HID or ISD SCT has greatly improved quality of life (QoL) levels post-HSCT compared to pre-HSCT. The occurrence of chronic graft versus host disease was the only identified adverse factor affecting each subscale of QoL. Physical and mental component summaries in adults as well as physical, mental, social, and role well-being in children were all similar between HID and ISD SCT at 5-year points. At the last followup, the proportion of returning to society was comparable between the HID and ISD groups, showing 78.0% versus 84.6% among children and 74.6% versus 81.2% among adults. These data suggest that haploidentical transplant can be considered a potential therapeutic option in the upfront setting for SAA patients in the absence of an HLA identical related or unrelated donor.

The role of donor type and pre‐transplant immunosuppression on outcomes of hematopoietic stem cell transplantation in children and young adults with severe aplastic anemia

“…Consequently, it is imperative to have options, other than matched unrelated donor HSCT available for minority populations with SAA. Our study results indicate that haplo‐HSCT with PTCy is safe and feasible for use in pediatric patients with SAA and, together with other published data, support including haploidentical donors in trials of upfront treatment for SAA in children, randomizing patients to IST versus HSCT 12–14 …”

Mesenchymal stromal cells plus basiliximab improve the response of steroid-refractory acute graft-versus-host disease as a second-line therapy: a multicentre, randomized, controlled trial

“…During allo-HSCT, HLA typing, donor selection, infection and GVHD prevention, and supportive care were performed according to our previous reports [27][28][29]. For patients with malignant haematologic diseases, the BU/CY-based conditioning protocol comprised the following: (1) in HID HSCT: cytarabine (4 g/m 2 per day on days −10 to −9); busulfan (3.2 mg/kg per day on days −8 to −6); cyclophosphamide (1.8 g/m 2 per day on days −5 to −4); methyl chloride hexamethylene urea nitrate (Me-CCNU, 250 mg/m 2 per day on day −3); thymoglobulin (ATG, 2.5 mg/kg per day on days −5 to −2, Sang Stat, Lyon, France); (2) in unrelated donor (URD) HSCT: cytarabine (2 g/m 2 per day on days −10 to −9); other regimens were the same as type (1); (3) in matched identical sibling donor (MSD) HSCT: hydroxycarbamide (80 mg/ kg) orally on day −10; cytarabine (2 g/m 2 per day on day −9); with ATG (1.5 mg/kg per day on days −5 to −3 for patients ≥40 years).…”

The Use of an Iterative Strategy of Cognitive Interview and Expert Consultation to Revise the Quality of Life Scale for Patients with Aplastic Anemia (QLS-AA)

“…Studies have shown that the 5-year survival rate of patients with severe aplastic anemia treated with HSCT or IST can reach more than 80%, while the 9-year survival rate of patients treated with HSCT is as high as 87–89%. 7 However, aplastic therapy is often accompanied by side effects such as decreased physical function, fatigue, diarrhea, nausea and vomiting, oral ulcer, hair loss, rash and respiratory problems. In addition, patients face a variety of risks such as disease recurrence, secondary clonal disease, infection, graft-versus-host disease (GVHD) and so on.…”