Cyclophosphamide-Based In Vivo T-Cell Depletion for HLA-Haploidentical Transplantation in Fanconi Anemia

Thakar, Monica S.; Bonfim, Carmem; Sandmaier, Brenda M.; O’Donnell, Paul V.; Ribeiro, Lisandro; Gooley, Ted; Deeg, H. Joachim; Flowers, Mary E.D.; Pasqüini, Ricardo; Storb, Rainer; Woolfrey, Ann E.; Kiem, Hans Peter

doi:10.3109/08880018.2012.708708

Cited by 28 publications

(25 citation statements)

References 28 publications

(34 reference statements)

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…While this alkylator-based approach to haploidentical alloHCT is particularly challenging for FA patients because of the patient’s underlying hypersensitivity to DNA cross-linking agents relative to the insensitive donor alloreactive T cells, there has been some success. In 3 patients, Thacker et al [67] have used haploidentical donor grafts followed by PTCy at 25 mg/kg on days +3 and +4 (rather than 50 mg/kg x 2 in other patient populations). All 3 patients engrafted with one dying at day +27 of disseminated toxoplasmosis and CMV.…”

Section: Introductionmentioning

confidence: 99%

Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations

Ebens

MacMillan

Wagner

2016

Expert Review of Hematology

View full text Add to dashboard Cite

Section: Introductionmentioning

confidence: 99%

Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations

Ebens

MacMillan

Wagner

2016

Expert Review of Hematology

View full text Add to dashboard Cite

“…Use of PT/Cy as GVHD prophylaxis, with or without additional immunosuppression, has been used with alternative donor sources in hematologic malignancies, leading to successful engraftment, as well as rates of GVHD, TRM, and graft failure comparable to HLA-matched related donors [10-12], without increased risk of posttransplant lymphoproliferative disorder [13]. Importantly, HSCT using alternative donors and PT/Cy has been performed for hemoglobinopathies, with GVHD and TRM comparable to that seen with HLA-matched related donors [14, 15], but reports using this strategy for other nonmalignant pediatric disorders, such as immunodeficiencies and bone marrow failure syndromes, are limited [16, 17]. Herein, we describe our institutional experience treating eleven pediatric patients with life-threatening, nonmalignant conditions using alternative donor sources, RIC, and PT/Cy for GVHD prophylaxis.…”

Section: Introductionmentioning

confidence: 99%

Alternative-Donor Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide for Nonmalignant Disorders

Klein¹,

Chen²,

Gamper³

et al. 2016

Biology of Blood and Marrow Transplantation

View full text Add to dashboard Cite

Allogeneic (allo-) hematopoietic stem cell transplant (HSCT) is curative for many nonmalignant pediatric disorders, including hemoglobinopathies, bone marrow failure syndromes, and immunodeficiencies. There is great success using HLA-matched related donors for these patients; however, the use of alternative donors has been associated with increased graft failure, graft versus host disease (GVHD), and transplant-related mortality (TRM). HSCT using alternative donors with post-transplantation cyclophosphamide (PT/Cy) for GVHD prophylaxis has been performed for hematologic malignancies with engraftment, GVHD, and TRM comparable to that seen with HLA-matched related donors. There are limited reports of HSCT in nonmalignant pediatric disorders other than hemoglobinopathies using alternative donors and PT/Cy. We transplanted eleven pediatric patients with life-threatening nonmalignant conditions using reduced intensity conditioning (RIC), alternative donors, and PT/Cy alone or in combination with tacrolimus and mycophenolate mofetil. We observed limited GVHD, no TRM, and successful engraftment sufficient to eliminate manifestations of disease in all patients. Allo-HSCT using alternative donors and PT/Cy shows promise for curing nonmalignant disorders; development of prospective clinical trials to confirm these observations is warranted.

show abstract

“…43 Simultaneously, this platform is a foundation for the recent treatment-optimization strategies, including thymic shielding to decrease the risk of opportunistic infections via enhanced immune reconstitution, haplo-HSCT with PT/CY, and de-escalation of TBI. 43,57,64,65 Results of alternative donor transplants for FA are shown in Table 4.…”

Section: Fanconi Anemiamentioning

confidence: 99%

Alternative donor transplant of benign primary hematologic disorders

Tolar

Sodani²,

Symons

2015

Bone Marrow Transplant

View full text Add to dashboard Cite

Hematopoietic SCT is currently the only curative therapy for a range of benign inherited and acquired primary hematologic disorders in children, including BM failure syndromes and hemoglobinopathies. The preferred HLA-matched sibling donor is available for only about 25% of such children. However, there has been substantial progress over the last four decades in the use of alternative donors for those without a matched sibling—including HLA-matched unrelated donors, HLA-haploidentical related donors and unrelated-donor umbilical cord blood—so that it is now possible to find a donor for almost every child requiring an allograft. Below, we summarize the relative merits and limitations of the different alternative donors for benign hematologic conditions, first generally, and then in relation to specific disorders, and suggest recommendations for selecting such an alternative donor.

show abstract

Cyclophosphamide-Based In Vivo T-Cell Depletion for HLA-Haploidentical Transplantation in Fanconi Anemia

Cited by 28 publications

References 28 publications

Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations

Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations

Alternative-Donor Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide for Nonmalignant Disorders

Alternative donor transplant of benign primary hematologic disorders

Contact Info

Product

Resources

About