Unrelated cord blood transplantation for newly diagnosed patients with severe acquired aplastic anemia using a reduced-intensity conditioning: high graft rejection, but good survival

Abstract

We report a single-center experience in treating 18 consecutive patients with severe aplastic anemia (SAA) who received unrelated cord blood transplantation (CBT). The median age was 17 years (range 5 --61 years). Sixteen cases received a reducedintensity regimen composed of CY (total dose1200 mg/m 2 ), rabbit antithymocyte globulin (ATG, total dose 30 mg/kg) and fludarabine (FLU, total dose 120 mg/m 2 ). CYA and mycophenolate mofetil were used as GVHD prophylaxis. Two patients were not evaluable for engraftment because of early death on day þ 21 and þ 22. Only one of the sixteen cases achieved engraftment, but experienced secondary graft failure 3 months post transplantation. Fifteen patients experienced primary graft rejection, but all of them acquired autologous recovery. The 3-month and 6-month cumulative incidence of response was 56% and 81%, respectively. So far, 16 patients have survived for 330 --1913 days (median, 750 days) after transplantation. The probability of OS at 2 years was 88.9%. Our data indicate that CBT for newly diagnosed SAA using no irradiation but FLU and ATG-based conditioning still seems to inevitably lead to the high risk of rejection, but may facilitate autologous recovery and improve survival with low risk of transplant-related mortality.

Similar survival, but better quality of life after myeloablative transplantation using unrelated cord blood vs matched sibling donors in adults with hematologic malignancies

“…The supportive care regimen, including prophylaxis for infection, G-CSF use and blood product infusion, was the same as previously reported. 23 …”

“…23 Corticosteroid-based treatment was considered when pre-engraftment syndrome (PES) and grade II or higher acute GVHD (aGVHD) occurred (0.5-2 mg/kg). If the manifestations of GVHD in any organ worsened over 3 days of treatment or if the skin did not improve by 5 days, second-line treatments were used, such as anti-CD25 monoclonal antibody (basiliximab), methotrexate and mesenchymal stem cells.…”

Allogeneic hematopoietic cell transplantation for severe aplastic anemia: similar long-term overall survival after transplantation with related donors compared to unrelated donors

“…Allogeneic HCT for SAA with an umbilical cord as graft source is generally not recommended if a related or an unrelated donor is available because of high risk of graft rejection, transplantation-related mortality, infectious complications and GVHD. [5][6][7][8][9] The conditioning regimen based on cyclophosphamide and ATG and the use of bone marrow as donor graft still seem to be an optimal setting for allogeneic HCT for SAA.…”

Pilot trial of risk‐adapted cyclophosphamide intensity based conditioning and HLA matched sibling and unrelated cord blood stem cell transplantation in newly diagnosed pediatric and adolescent recipients with acquired severe aplastic anemia

“…The largest series of UCBT for refractory SAA in 71 adults and pediatric patients from 23 EBMT centers showed a 51% engraftment rate and 38% 3‐year OS, with a clear association of outcome with TNC dose . Moving from refractory disease to upfront treatment, a series of eighteen adult patients with newly diagnosed SAA treated with UCBT had an encouraging 2‐year survival of 88.9% . This OS included an extremely high incidence of graft failure (16/18 patients) with the hypothesis that the immunosuppressive effect of the combination of T cell‐directed conditioning regimen with autologous reconstitution mitigated the impact of graft failure on OS .…”