Abstract
This retrospective study explored the incidence of autoimmune cytopenia (AIC) in 530 paediatric and adult patients with acquired aplastic anaemia (aAA) who underwent first allogeneic HSCT between 2002 and 2012. AIC was a rare complication with a cumulative incidence of AIC at 1, 3, 5 and 10 years post HSCT of 2.5% (1.2–3.9 95% CI), 4.4% (2.6–6.2 95% CI), 4.6% (2.8–6.5 95% CI) and 5.1% (3.1–7.2 95% CI). Overall survival at 5 years after diagnosis of AIC was 85.9% (71–100 95% CI). Twenty-five patients were diagnosed with AIC at a median of 10.6 (2.6–91.5) months post HSCT. Eight (32%) patients were diagnosed with immune thrombocytopenia (ITP), seven (28%) with autoimmune haemolytic anaemia (AIHA), seven (24%) with Evans syndrome and four (16%) with autoimmune neutropenia (AIN). Treatment strategies were heterogeneous. Complete responses were seen in 12 of 25 patients, with death in three patients. In multivariable Cox analysis of a subgroup of 475 patients, peripheral blood stem cell (PBSC) transplant was associated with higher risk of AIC compared with bone marrow (BM) when conditioning regimens contained fludarabine and/or alemtuzumab (2.81 [1.06–7.49 95% CI]; p = 0.038), or anti-thymocyte globulin (ATG) (2.86 [1.11–7.37 95% CI]; p = 0.029). Myeloablative conditioning was associated with a lower risk of AIC compared with reduced intensity conditioning (RIC) in fludarabine and/or alemtuzumab (0.34 [0.12–0.98 95% CI]; p = 0.046) and ATG containing regimens (0.34 [0.12–0.95 95% CI]; p = 0.04). These findings provide clinically useful information regarding the incidence of a rare and potentially life-threatening complication of allogeneic HSCT for aAA, and further support for BM as the preferred stem cell source for transplant of patients with aAA.
Highlights
Graft versus host disease (GvHD) describes alloreactivity between a transplanted donor immune system and host major or minor histocompatibility antigen, and is the principal immune mediated complication of allogeneic haematopoietic stem cell transplant (HSCT)
Acquired aplastic anaemia is thought to be an autoimmune disorder in most cases [10] and we hypothesised that the incidence of post HSCT autoimmune cytopenias (AIC) in this population may be higher than described in other patient populations
bone marrow examination (BME) was not performed in two patients diagnosed with immune thrombocytopenia (ITP)
Summary
Graft versus host disease (GvHD) describes alloreactivity between a transplanted donor immune system and host major or minor histocompatibility antigen, and is the principal immune mediated complication of allogeneic haematopoietic stem cell transplant (HSCT). Reported transmission of autoantibodies and AIDs from donor to recipient, and a case of immune thrombocytopenia (ITP) in the context of syngeneic HSCT suggest that this is a pathological entity distinct from GvHD [1, 2]. The most commonly encountered AIDs post HSCT are autoimmune cytopenias (AIC) [3]. Acquired aplastic anaemia (aAA) is thought to be an autoimmune disorder in most cases [10] and we hypothesised that the incidence of post HSCT AIC in this population may be higher than described in other patient populations. We sought to explore AIC diagnostic strategies, treatment approaches and patient outcomes across European Society for Blood and Marrow Transplantation (EBMT) centres
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