Immunosuppressive therapy versus haploidentical transplantation in adults with acquired severe aplastic anemia

Abstract

Our study aimed to compare treatment outcomes between hematopoietic stem cell transplantation (HSCT) from haploidentical donors (HID) and immunosuppressive therapy (IST) in adults with acquired severe aplastic anemia (SAA). The medical records of 113 SAA adults who received IST, including rabbit ATG and cyclosporin (N = 37), or HID HSCT (N = 76) within 6 months of diagnosis at two institutions were retrospectively reviewed. Estimated 8-year overall survival (OS) was comparable between the IST and HID HSCT groups (75.6 vs. 83.7%, respectively, P = 0.328), but failure free survival (FFS) was significantly lower in IST group than HID HSCT group (38.5 vs. 83.7%, respectively, P = 0.001). Furthermore, a significant improvement in FFS was observed with HSCT over IST in patients under 40 years old. At the last follow-up, patients in HSCT group achieved better Karnofsky Performance Status (KPS) than those in IST group (100 [20-100] vs. 90 [20-100], P = 0.002). In terms of blood count, 83.1% (54/65) of patients in HSCT group showed complete recovery compared to only 38.2% (13/34) in IST group (P < 0.001). These data suggest that HID HSCT could be an effective alternative treatment option for SAA adults, and additional prospective studies are necessary.

Comparable survival outcome between transplantation from haploidentical donor and matched related donor or unrelated donor for severe aplastic anemia patients aged 40 years and older: A retrospective multicenter cohort study

“…5 Our previous study also showed a trend of higher FFS in patients older than 40 years of age in 8th year after HID-HSCT compared with those only received IST. 26 Based on these results, it is reasonable for selected patients to undergo HSCT to get complete hematopoietic recovery and favorable survival rate when facing the choice between HSCT and IST, with no need to be concerned about pros and cons of grafts from MSD, URD, or even HID.…”

Allo-HSCT compared with immunosuppressive therapy for acquired aplastic anemia: a system review and meta-analysis

“…All studies reported the outcomes of AA patients treated with first-line allo-HSCT or IST [11][12][13][14][15][16][17][18][19][20][21][22][23][24][25]. The allo-HSCT group was divided into two subgroups: BMT-MRD and BMT-MUD.…”

A Child With Pancytopenia and Optic Disc Swelling

“…In a recent study, success was demonstrated in nonrefractory SAA with haploidentical HSCT without initial IST. 25 More data exist for haploidentical HSCT in the treatment of refractory SAA for patients without an MSD. 26,27 In 1 study, researchers demonstrated that 94% of patients achieved successful engraftment with a 3-year survival of 89%, 28 with similar findings in a registry-based comparison.…”