Hematopoieticstem cell transplantation (HSCT) is safe and efficient in children with Diamond-Blackfananemia (DBA), especially in the absence of a matched sibling donor (MSD) ormatched unrelated donor (UD), according to a study published in BloodAdvances.

DBAis associated with congenital anomalies and cancer predisposition. Treatmentfor DBA includes regular transfusions with chelation and corticoid therapies. Patientsthat remain transfusion dependent are at risk of complications due to ironoverload and rigorous chelation therapy is indicated. HSCT is mainly utilizedin severe DBA cases and those with secondary myelodysplastic syndrome, yetrecent studies suggest HSCT may also be useful to correct the hematologicalphenotype in children and is indicated in transfusion-dependent patients.

Ateam of European researchers conducted an analysis to determine survivaloutcomes in patients with DBA who underwent allogeneic HSCT. Data was collectedfrom 70 children with DBA (<18 years; 44 males) who were registered inGermany or France between 1985 and 2017.

Primary outcomes included engraftment, cumulative incidence of acute graft-versus-host disease (aGVHD) and chronic graft-versus-host disease (cGVHD), probability of overall survival (pOS), and the probability of cGVHD-free survival (cGFS).

Medianage at HSCT was 5.5 years, and 26% of patients were aged 10 years or younger atthe time of transplantation. A large number of transplantations (64%) wereperformed from an MSD and most procedures were performed after 1999 (73%). Allpatients achieved primary engraftment.

Thecumulative incidence of grade 2 to grade 4 and grade 3 to grade 4 aGVHD was 24%and 7%, respectively. Furthermore, the cumulative incidence of cGVHD was 11%. Nosignificant difference in the incidence of aGVHD comparing MSD and UD HSCT wasobserved. Over time, the probability of cGFS (87%) significantly improved from68% in years earlier than 2000 compared with 94% in 2000 and later (P <.01). cGFS was comparable forpatients who received a transplant from either a MSD or an unrelated donor(UD). However, after 1999, no severe cGVHD or deaths were reported followingMSD-HSCT, whereas the cGFS for 24 patients transplanted from an UD was 87%. Thechange of cGFS in patients transplanted at a younger age compared with olderpatients (<10 years: 90% vs 10-18 years: 78%) was not statisticallysignificant.

Studylimitations included the inability to fully analyze the role of ATG and ATGdosing due to the retrospective nature of the study, the lack of availabilityof genetic testing for all of the patients, and the small sample size ofpatients between the ages of 10 to 18 years.

In summary,these data indicate that HSCT is efficient and safe in young DBA patients and should be considered ifa MSD or matched UD is available. HSCT for transfusion dependency only must becritically discussed in older patients, the authors concluded.

Reference

Strahm B, Loewecke F, Niemeyer CM, et al. Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia. Blood Adv. 2020;4(8):17601769.

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Outcomes of Hematopoietic Stem Cell Transplantation in Children With Diamond-Blackfan Anemia - Hematology Advisor