Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program

Abstract

Multivariate proportional hazards regression was used to compare outcome by donor type and identify other prognostic factors. Most transplant recipients were younger than 5 years (79%), had a pretransplantation performance score greater than or equal to 90% (63%), received pretransplantation preparative regimens without radiation (82%), and had non-Tcell-depleted grafts (77%). Eighty percent received their transplant after 1986. The 5-year probability of survival (95% confidence interval) for all subjects was 70% (63%-77%). Probabilities differed by donor type: 87% (74%-93%) with HLAidentical sibling donors, 52% (37%-65%) with other related donors, and 71% (58%-80%) with unrelated donors (P ‫؍‬ .0006).

Allogeneic hematopoietic stem cell transplantation for seven children with X‐linked hyper‐IgM syndrome: A single center experience

“…In the previous reports, T-cell depletion or CD34-positive selection was often performed in order to prevent GVHD when unrelated donor marrow was selected [18,20]. However, we did not use either method for our patients because they are sometimes associated with a risk of graft rejection and EBV-associated posttransplant lymphoproliferative disease (PTLD) when transplanting the immunodeficiency patients [11]. As GVHD in our series were relatively well controlled except for one case (Patient 2), we believe that HLAmatched unrelated donor marrow transplant can be safely performed without T-cell depletion or CD34-positive selection.…”

“…Another patient also suffered from pre-transplant complications involving the lung, but the failure was due to severe post-transplantation infection complicated with secondary graft failure and grade 4 acute GVHD. It has been observed that older patients with primary immunodeficiency disorders tend to be at higher risk for HSCT [11,20]. The reason for this is that they may have more severe pre-transplant infections and may have serious damage in critical organs such as lung and liver because of recurrent or persistent infections.…”

Successful Haploidentical Transplantation with High Doses of CD34+-Selected Peripheral Blood Stem Cells in Wiskott-Aldrich Syndrome: A Case Report

“…Apart from their case, one more unmanipulated haplo-identical transplant was reported. There is a 4-5-fold increase in mortality after haploidentical compared to HLA-identical sibling grafts [7,11]. These reports show a high incidence of graft failure and poor immune reconstitution following T cell-depleted haploidentical HSCTs.…”

“…Therefore, it is prudent to monitor levels and target the dose to achieve a steady state concentration of greater than 200 ng/ml to assure engraftment. It is determined that patient age, disease severity, and splenectomy did not affect outcome in HSCT of WAS patients in which HLA-identical sibling marrow grafts were used [7,8].…”

Functional correction of T cells derived from patients with the Wiskott–Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein

“…[3][4][5] However, most patients lack matched sibling donors, and transplant using matched unrelated donors is effective only in a subset of patients. 6 Alternative methods, such as gene therapy, therefore warrant exploration.…”