Browsing by Author "Fasth, Anders"
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Item Open Access Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism.(Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2012-09) Eapen, Mary; Ahn, Kwang Woo; Orchard, Paul J; Cowan, Morton J; Davies, Stella M; Fasth, Anders; Hassebroek, Anna; Ayas, Mouhab; Bonfim, Carmem; O'Brien, Tracey A; Gross, Thomas G; Horwitz, Mitchell; Horwitz, Edwin; Kapoor, Neena; Kurtzberg, Joanne; Majhail, Navneet; Ringden, Olle; Szabolcs, Paul; Veys, Paul; Baker, K ScottIt is uncertain whether late mortality rates after hematopoietic cell transplantation for severe combined immunodeficiency (SCID), non-SCID primary immunodeficiency diseases (non-SCID PIDD), and inborn errors of metabolism (IEM) return to rates observed in the general population, matched for age, sex, and nationality. We studied patients with SCID (n = 201), non-SCID PIDD (n = 405), and IEM (n = 348) who survived for at least 2 years after transplantation with normal T cell function (SCID) or >95% donor chimerism (non-SCID PIDD and IEM). Importantly, mortality rate was significantly higher in these patients compared with the general population for several years after transplantation. The rate decreased toward the normal rate in patients with SCID and non-SCID PIDD beyond 6 years after transplantation, but not in patients with IEM. Active chronic graft-versus-host disease at 2 years was associated with increased risk of late mortality for all diseases (hazard ratio [HR], 1.87; P = .05). In addition, late mortality was higher in patients with non-SCID PIDD who received T cell-depleted grafts (HR 4.16; P = .007) and in patients with IEM who received unrelated donor grafts (HR, 2.72; P = .03) or mismatched related donor grafts (HR, 3.76; P = .01). The finding of higher mortality rates in these long-term survivors for many years after transplantation confirms the need for long-term surveillance.Item Open Access Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita.(Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2013-08) 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 AWe 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.Item Open Access Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.(Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2011-09) Ruggeri, Annalisa; Eapen, Mary; Scaravadou, Andromachi; Cairo, Mitchell S; Bhatia, Monica; Kurtzberg, Joanne; Wingard, John R; Fasth, Anders; Lo Nigro, Luca; Ayas, Mouhab; Purtill, Duncan; Boudjedir, Karim; Chaves, Wagnara; Walters, Mark C; Wagner, John; Gluckman, Eliane; Rocha, Vanderson; Eurocord Registry; Center for International Blood and Marrow Transplant Research; New York Blood CenterWe examined the efficacy of unrelated cord blood (CB) transplantation in children with thalassemia (n = 35) and sickle cell disease (n = 16), using data reported to 3 registries. Donor-recipient pairs were matched at HLA-A and -B (antigen level) and DRB1 (allele level) in 7 or HLA mismatched at 1 (n = 18), 2 (n = 25), or 3 loci (n = 1). Transplant conditioning was myeloablative (n = 39) or reduced intensity (n = 12). Neutrophil recovery with donor chimerism was documented in 24 patients; 11 patients developed grade II-IV acute graft-versus-host disease (aGVHD) and 10 patients, chronic GVHD (cGVHD). Overall survival (OS) and disease-free survival (DFS) were 62% and 21% for thalassemia and 94% and 50% for sickle cell disease (SCD), respectively. In multivariate analysis, engraftment rate (hazard ratio [HR] 2.2, P = .05) and DFS (HR 0.4, P = .01) were higher with cell dose >5 × 10(7)/kg. The 2-year probability of DFS was 45% in patients who received grafts with cell dose >5 × 10(7)/kg and 13% with lower cell dose. Primary graft failure was the predominant cause of treatment failure occurring in 20 patients with thalassemia and 7 patients with SCD. Primary graft failure was fatal in 5 patients with thalassemia. These results suggest that only CB units containing an expected infused cell dose >5 × 10(7)/kg should be considered for transplantation for hemoglobinopathy.