Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation

Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation

Fioredda, F. Blood. 2015 (Link to abstract)                                 


This multicenter, retrospective study included patients from the EBMT and SCETIDE registries. 136 Severe Congenital Neutropenia (SCN) patients underwent HSCT between 1990-2012 at European and Middle Eastern centers. Indications leading to transplant included: HD-GCSF requirements (79%), severe infection (37%), transformation to MDS before HSCT (16%).


The following complications were seen: Graft failure: Probability after 90 days from HSCT was 10%, probability of engraftment was 92% in HLA-matched, and 72% in mismatched donor. GVHD: Cumulative incidence at 90 days (grade 2-4) was 21%. 2 factors associated with a significant lower rate of GVHD: HLA-matched related donor and double prophylaxis with CSA and MTX. Peripheral blood as sources of HSCT was associated with higher cGvHD incidence.


Overall Survival: 3-year OS after HSCT 82%. Median time from transplant to death: 3 months, with 80% of the deaths occurring within 12 months after transplant. [NW4] Transplants undertaken in later years had better survival (OS-3ys 66% in 1990-2000, 79% 2001-2007 and 91% in 2008-2012). No secondary malignancies were reported.


* HSCT is an option for treatment of SCN with an acceptable 3 year OS and EFS (82% and 71%, respectively). TRM is 17% and therefore appropriate selection of patients and SC sources as well as GVHD prophylaxis are important factors.