Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita.

Gadalla, Shahinaz M

Sales-Bonfim, Carmem

Carreras, Jeanette

Alter, Blanche P

Antin, Joseph H

Ayas, Mouhab

Bodhi, Prasad

Davis, Jeffrey

Davies, Stella M

Deconinck, Eric

Deeg, H Joachim

Duerst, Reggie E

Fasth, Anders

Ghavamzadeh, Ardeshir

Giri, Neelam

Goldman, Frederick D

Kolb, E Anders

Krance, Robert

Kurtzberg, Joanne

Leung, Wing H

Srivastava, Alok

Or, Reuven

Richman, Carol M

Rosenberg, Philip S

Toledo Codina, Jose Sanchez de

Shenoy, Shalini

Socié, Gerard

Tolar, Jakub

Williams, Kirsten M

Eapen, Mary

Savage, Sharon A

2022-03-23T20:23:58Z

2022-03-23T20:23:58Z

2013-08

2022-03-23T20:23:58Z

We describe outcomes after allogeneic transplantation in 34 patients with dyskeratosis congenita who underwent transplantation between 1981 and 2009. The median age at transplantation was 13 years (range, 2 to 35). Approximately 50% of transplantations were from related donors. Bone marrow was the predominant source of stem cells (24 of 34). The day-28 probability of neutrophil recovery was 73% and the day-100 platelet recovery was 72%. The day-100 probability of grade II to IV acute GVHD and the 3-year probability of chronic graft-versus-host disease were 24% and 37%, respectively. The 10-year probability of survival was 30%; 14 patients were alive at last follow-up. Ten deaths occurred within 4 months from transplantation because of graft failure (n = 6) or other transplantation-related complications; 9 of these patients had undergone transplantation from mismatched related or from unrelated donors. Another 10 deaths occurred after 4 months; 6 of them occurred more than 5 years after transplantation, and 4 of these were attributed to pulmonary failure. Transplantation regimen intensity and transplantations from mismatched related or unrelated donors were associated with early mortality. Transplantation of grafts from HLA-matched siblings with cyclophosphamide-containing nonradiation regimens was associated with early low toxicity. Late mortality was attributed mainly to pulmonary complications and likely related to the underlying disease.

S1083-8791(13)00234-6

1083-8791

1523-6536

https://hdl.handle.net/10161/24684

eng

Elsevier BV

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

10.1016/j.bbmt.2013.05.021

Humans

Dyskeratosis Congenita

Treatment Outcome

Hematopoietic Stem Cell Transplantation

Transplantation, Homologous

Survival Analysis

Retrospective Studies

Adolescent

Adult

Child

Child, Preschool

Female

Male

Young Adult

Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita.

Journal article

Kurtzberg, Joanne|0000-0002-3370-0703

1238

1243

8

Duke

School of Medicine

Clinical Science Departments

Institutes and Centers

Pathology

Pediatrics

Duke Cancer Institute

Institutes and Provost's Academic Units

Initiatives

Duke Innovation & Entrepreneurship

Pediatrics, Transplant and Cellular Therapy

Published

19