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.