Safe and Efficacious Allogeneic Bone Marrow Transplantation for Nonmalignant Disorders Using Partial T Cell Depletion and No Posttransplantation Graft-Versus-Host-Disease Prophylaxis

Elhasid, Ronit; Arush, Myriam Ben; Zaidman, Irena; Leiba, Ronit; Barak, Asher; Postovsky, Sergey; Haddad, Nuhad; Katz, Tamar; Pollack, Shimon; Sami, Ivanka; Gidoni, O.; Rubin, Dina; Mandel, Hanna; Attias, Dina; Reisner, Yaīr; Rowe, Jacob M.

doi:10.1016/j.bbmt.2006.10.028

Cited by 12 publications

(9 citation statements)

References 27 publications

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“…Five patients with class I thalassemia received BU, CY, anti-thymocyte globulin, fludarabine and moderate T-cell depletion, as described elsewhere. 6 Six patients with class III thalassemia received protocol P26 7 using hydroxyurea and azathioprine and later, fludarabine, CY and BU. GVHD prophylaxis, the number of CD34 þ cells per kg infused and the blood groups of donor and recipient are included in Tables 1 and 2.…”

Section: Patientsmentioning

confidence: 99%

Glutathione S-transferase T1-null seems to be associated with graft failure in hematopoietic SCT

Elhasid

Krivoy

Rowe

et al. 2010

Bone Marrow Transplant

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Section: Patientsmentioning

confidence: 99%

Glutathione S-transferase T1-null seems to be associated with graft failure in hematopoietic SCT

Elhasid

Krivoy

Rowe

et al. 2010

Bone Marrow Transplant

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“…Fifteen patients with class 3 thalassemia received a conditioning regimen using protocol P26 [12], four patients received protocol P26.1 for a second HSCT [24], and two patients received a protocol using partial T cell depletion as GVHD prophylaxis [25].…”

Section: Conditioning Regimens and Gvhd Prophylaxismentioning

confidence: 99%

“…This protocol included Bu 16mg/kg, Cy 200mg/kg, ATG 25mg/kg, and Flu 200mg/m 2 with no GVHD prophylaxis post-transplantation [25].…”

Section: Eight Patients Received Grafts From Msd Using Partial T-cellmentioning

confidence: 99%

Allogeneic Stem Cell Transplantation in Congenital Hemoglobinopathies Using a Tailored Busulfan-Based Conditioning Regimen: Single-Center Experience

Zaidman

Rowe

Khalil

et al. 2016

Biology of Blood and Marrow Transplantation

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Hematopoietic stem cell transplantation (HSCT) is the only proven curative option for patients with hemoglobinopathies, both thalassemia and sickle cell anemia (SCA). A busulfan-based myeloablative conditioning regimen is the standard of care for HSCT in these patients, although increased treatment-related morbidity, including veno-occlusive disease (VOD), has been demonstrated. Thirty-eight pediatric patients, median age 8 years (range, 6 months to 22 years), suffering from hemoglobinopathy were treated at Rambam Medical Center in Haifa, Israel, between 1998 and 2011. Thirty-four patients had thalassemia major and 4 had SCA. The 38 patients underwent 40 HSCTs, 34 of which were first transplants and 6 second transplants. Most transplants (32/40) were from matched sibling donors. Sources of stem cells were peripheral blood in 30 transplants, bone marrow in 7 transplants, and cord blood in 3 transplants. All received different customized busulfan-based conditioning regimens tailored by pharmacokinetic analysis of busulfan levels. Primary engraftment occurred in 37 of 40 transplants. Neutrophil engraftment (>.5 × 10(9)/L) occurred at a median of 15.3 days post-transplantation (range, 10 to 45). Platelet transfusion independence (>20 × 10(9)/L) occurred at a median of 22.3 days (range, 11 to 60). The rate of 5-year overall survival for thalassemia patients after first transplantation was 90.5% ± 5.3%. The rate of 5-year thalassemia-free survival was 81.7% ± 6.8%. Cumulative incidence of acute graft-versus-host disease (GVHD) was 17.6%. Rate of grades III to IV GVHD was 8.8%. Cumulative incidence of chronic GVHD was 23.5%, with 11.8% incidence of extensive chronic GVHD. One patient developed VOD. Full donor chimerism occurred in 36.4% of patients with class 1 + 2 thalassemia, compared with 78.6% in class 3 thalassemia (P = .049). Overall survival above 90% in patients undergoing their first transplant was demonstrated using busulfan-based conditioning regimens. The low incidence of VOD was probably due to busulfan area under the curve measurements and dose adjustment.

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“…A safe and effective conditioning protocol has been developed in Israel [38], but no lupus patients were transplanted. A large number of SLE patients were allotransplanted in Ahmedabad according to a complicated conditioning protocol [39], but they all relapsed after a mean of 7.35 months of disease-free interval.…”

Section: Allogeneic Transplantationmentioning

confidence: 99%

Hematopoietic Stem Cell Transplantation for Systemic Lupus Erythematosus

Champ

2012

Clinical and Developmental Immunology

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Two streams of research are at the origin of the utilization of hematopoietic stem cell transplantation (HSCT) for severe autoimmune diseases (SADs). The allogeneic approach came from experimental studies on lupus mice, besides clinical results in coincidental diseases. The autologous procedure was encouraged by researches on experimental neurological and rheumatic disorders. At present the number of allogeneic HSCT performed for human SADs can be estimated to not over 100 patients, and the results are not greatly encouraging, considering the significant transplant-related mortality (TRM) and the occasional development of a new autoimmune disorder and/or relapses notwithstanding full donor chimerism. Autologous HSCT for refractory SLE has become a major target. Severe cases have been salvaged, TRM is low and diminishing, and prolonged clinical remissions are obtainable. Two types of immune resetting have been established, “re-education” and regulatory T cell (Tregs) normalization. Allogeneic HSCT for SLE seems best indicated for patients with disease complicated by an oncohematologic malignancy. Autologous HSCT is a powerful salvage therapy for otherwise intractable SLE. The duration of remission in uncertain, but a favorable response to previously inactive treatments is a generally constant feature. The comparison with new biological agents, or the combination of both, are to be ascertained.

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Safe and Efficacious Allogeneic Bone Marrow Transplantation for Nonmalignant Disorders Using Partial T Cell Depletion and No Posttransplantation Graft-Versus-Host-Disease Prophylaxis

Cited by 12 publications

References 27 publications

Glutathione S-transferase T1-null seems to be associated with graft failure in hematopoietic SCT

Glutathione S-transferase T1-null seems to be associated with graft failure in hematopoietic SCT

Allogeneic Stem Cell Transplantation in Congenital Hemoglobinopathies Using a Tailored Busulfan-Based Conditioning Regimen: Single-Center Experience

Hematopoietic Stem Cell Transplantation for Systemic Lupus Erythematosus

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