Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients.
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Hashem Hasan, Bucciol Giorgia, Ozen Seza, Unal Sule, Bozkaya Ikbal Ok, Akarsu Nurten, Taskinen Mervi, Koskenvuo Minna, Saarela Janna, Dimitrova Dimana, Hickstein Dennis D, Hsu Amy P, Holland Steven M, Krance Robert, Sasa Ghadir, Kumar Ashish R, Müller Ingo, de Sousa Monica Abreu, Delafontaine Selket, Moens Leen, Babor Florian, Barzaghi Federica, Cicalese Maria Pia, Bredius Robbert, van Montfrans Joris, Baretta Valentina, Cesaro Simone, Stepensky Polina, Benedicte Neven, Moshous Despina, Le Guenno Guillaume, Boutboul David, Dalal Jignesh, Brooks Joel P, Dokmeci Elif, Dara Jasmeen, Lucas Carrie L, Hambleton Sophie, Wilson Keith, Jolles Stephen, Koc Yener, Güngör Tayfun, Schnider Caroline, Candotti Fabio, Steinmann Sandra, Schulz Ansgar, Chambers Chip, Hershfield Michael, Ombrello Amanda, Kanakry Jennifer A, Meyts Isabelle
Аннотация
METHODS: We conducted a retrospective study on the outcome of HCT in patients with DADA2. The primary outcome was overall survival (OS).
RESULTS: Thirty DADA2 patients from 12 countries received a total of 38 HCTs. The indications for HCT were BMF, immune cytopenia, malignancy, or immunodeficiency. Median age at HCT was 9 years (range: 2-28 years). The conditioning regimens for the final transplants were myeloablative (n = 20), reduced intensity (n = 8), or non-myeloablative (n = 2). Donors were HLA-matched related (n = 4), HLA-matched unrelated (n = 16), HLA-haploidentical (n = 2), or HLA-mismatched unrelated (n = 8). After a median follow-up of 2 years (range: 0.5-16 years), 2-year OS was 97%, and 2-year GvHD-free relapse-free survival was 73%. The hematological and immunological phenotypes resolved, and there were no new vascular events. Plasma ADA2 enzyme activity normalized in 16/17 patients tested. Six patients required more than one HCT.
CONCLUSION: HCT was an effective treatment for DADA2, successfully reversing the refractory cytopenia, as well as the vasculopathy and immunodeficiency.
CLINICAL IMPLICATIONS: HCT is a definitive cure for DADA2 with > 95% survival.