Haematopoietic stem cell transplantation for hepatitis‐associated aplastic anaemia and non‐hepatitis‐associated aplastic anaemia: A propensity score‐matched analysis

Abstract

To validate the efficacy and safety of haematopoietic stem cell transplantation (HSCT) in hepatitis-associated aplastic anaemia (HAAA) patients, we reviewed 260 patients who underwent HSCT for acquired aplastic anaemia and eventually included 30 HAAA patients and 90 non-HAAA patients using propensity score matching. In the HAAA group, the estimated 5-year overall survival rate (75.8% vs. 86.5%, p = 0.409), failure-free survival (FFS) rate (74.0% vs. 83.2%, p = 0.485), graftversus-host disease (GVHD)-free FFS rate (61.2% vs. 67.6%, p = 0.669) after HSCT were slightly lower but not statistically significant than those in the non-HAAA group. Both groups did not significantly differ in engraftment, post-transplant severe infection, cytomegalovirus (CMV) or Epstein-Barr virus viraemia, or GVHD incidences. The patterns of immune reconstitution were broadly consistent between the two groups. When stratifying HAAA patients according to donor type, no significant differences in survival, transplant-related mortality, or GVHD cumulative incidences were observed. CMV viraemia (68.7% vs 8.3%, p = 0.009) occurred more commonly in haploidentical donor (HID) transplants than in matched sibling donor transplants. However, early CMV disease incidence (5.6% vs. 0.0%, p = 1.000) was low. Overall, the post-transplant outcomes of HAAA patients were comparable to those of non-HAAA patients after balancing potential confounders, and HID-HSCT can offer an alternative curative option for HAAA.

Addition of ruxolitinib to standard graft-versus-host disease prophylaxis for allogeneic stem cell transplantation in aplastic anemia patients

“…Unfortunately, the conditioning regimen is significantly different between the historical control group and the ruxolitinib group (Supplementary Table 1 ) whereby higher proportion of the historical cohort used ALG (64.47% vs 42.86) and FAC (39.47% vs 14.29%) as conditioning regimen. We thus performed propensity score matching [ 29 ] to minimize the effects of confounding factors resulting in 35 patients in the historical-matched-control group (hereafter refer to control group). The matched patients were not significantly different in terms of baseline characteristics compared to ruxolitinib group, which are shown in Table 2 .…”

“…Two differentially distributed variables i.e., conditioning regimens (regimen w/o Bu) and ATG types (rATG or pALG) were Included in the propensity score matching (PSM) analysis to identify patients from historical cohort to match the ruxolitinib cohort. A 1:1 nearest neighbor matching with a caliper width of 0.2 [ 29 ] with significance level at P < 0.05 were adopted.…”

Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantation

“…More recently, only one study conducted by the Japan Society for Haematopoietic Cell Transplantation 16 reported a 5-year overall survival (OS) rate of 86.0% and a 100-day cumulative incidence of grade II–IV acute graft-versus-host disease (aGVHD) at 11.1% among HAAA patients who received HSCT from HLA-identical donors. In our published article 4 , we conducted a comparative analysis, matching 30 patients who underwent HAAA procedures at our center with patients without HAAA. The findings showed that estimated 5-year OS rate (75.8% vs. 86.5%, P = 0.409) and FFS rate (74.0% vs. 83.2%, P = 0.485) after HSCT were slightly lower but not statistically significantly different from those in the non-HAAA group.…”

“…Its pathogenesis remains poorly understood. Previous studies 2 – 4 have shown that HAAA is more prevalent in adolescent males, and most patients do not exhibit evidence of active hepatitis A, B, C, D, E, G and Torque Teno virus during diagnosis. Both HAAA and non-viral hepatitis may share a common etiology, which is mainly T-cell mediated immune dysregulation.…”