Conditioning regimens in allo-SCT for thalassemia major

Savani, Bipin B.

doi:10.1038/bmt.2013.216

Cited by 15 publications

(9 citation statements)

References 32 publications

(47 reference statements)

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“…24 Conditioning regimens designed to improve transplant outcomes have shown suboptimal results due to high rejection and TRM rates. 11,[24][25][26] In an attempt to reduce transplant-related toxicity in class 3 patients, the cumulative dose of Cy in the standard BuCy regimen was reduced from 200 mg/kg to 160 to 120 mg/kg. 11 Although this approach significantly reduced TRM, it was associated with and increased rejection rate (up to 30%).…”

Section: Discussionmentioning

confidence: 99%

Optimal Outcomes in Young Class 3 Patients With Thalassemia Undergoing HLA-Identical Sibling Bone Marrow Transplantation

et al. 2016

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Section: Discussionmentioning

confidence: 99%

Optimal Outcomes in Young Class 3 Patients With Thalassemia Undergoing HLA-Identical Sibling Bone Marrow Transplantation

et al. 2016

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“…In the age group ,4 years of age, where Tt seems particularly important to reduce rejection, 15 we observed no significant rejection differences between the ATG and Tt groups. A combination of treosulfan, Tt, and fludarabine has also been advocated as the preferable BMT conditioning for patients with thalassemia, particularly those with higher transplantation risk [55][56][57] ; however, this recommendation is not based on prospective studies, and the combined use of 2 myeloablative alkylating agents may be associated with high infertility rates 20 as well as substantial increases in costs. A formal cost analysis could not be performed, but the average all-inclusive cost per BMT was ;12 000 US dollars and that of family support was ;2000 US dollars.…”

Section: Discussionmentioning

confidence: 99%

ATG vs thiotepa with busulfan and cyclophosphamide in matched-related bone marrow transplantation for thalassemia

et al. 2017

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Key Points• In low-risk BMT for thalassemia, ATGBuCy seems as effective as Tt-BuCy in the prevention of rejection and may decrease transplant-related mortality. received ATG-BuCy. All patients were ,15 years and had no hepatomegaly (liver #2 cm from costal margin). Actuarial overall survival in the Tt-BuCy and ATG-BuCy groups was 87% and 94% and thalassemia-free survival was 80% and 85% at a median follow-up of 37 and 17 months, respectively, with no significant differences by log-rank statistics. SubstitutingTt with ATG in the standard BuCy context seems safe and effective and may decrease transplant-related mortality. Higher fertility rates are expected for patients who received ATG-BuCy.

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“…For patients at high risk, conditioning needs to be titrated to reduce toxicity and at the same time, prevent graft rejection. Conditioning regimens for thalassemia have recently been reviewed [55]. Reduced intensity conditioning had generally had disappointing results due to autologous recovery [56].…”

Section: Bone Marrow Transplantationmentioning

confidence: 99%

Thalassemia 2016: Modern medicine battles an ancient disease

Rund

2015

American J Hematol

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Thalassemia was first clinically described nearly a century ago and treatment of this widespread genetic disease has greatly advanced during this period. DNA‐based diagnosis elucidated the molecular basis of the disease and clarified the variable clinical picture. It also paved the way for modern methods of carrier identification and prevention via DNA‐based prenatal diagnosis. Every aspect of supportive care, including safer blood supply, more regular transfusions, specific monitoring of iron overload, parenteral and oral chelation, and other therapies, has prolonged life and improved the quality of life of these patients. Significant advances have also been made in allogenic bone marrow transplantation, the only curative therapy. Recently, there has been a rejuvenated interest in studying thalassemia at the basic science level, leading to the discovery of previously unknown mechanisms leading to anemia and enabling the development of novel therapies. These will potentially improve the treatment of, and possibly cure the disease. Pathways involving activin receptors, heat shock proteins, JAK2 inhibitors and macrophage targeted therapy, among others, are being studied or are currently in clinical trials for treating thalassemia. Novel types of genetic therapies are in use or under investigation. In addition to the challenges of treating each individual patient, the longer survival of thalassemia patients has raised considerations regarding worldwide control of thalassemia, since prevention is not universally implemented. This review will trace a number of the original medical milestones of thalassemia diagnosis and treatment, as well as some of the most recent developments which may lead to innovative therapeutic modalities. Am. J. Hematol. 91:15–21, 2016. © 2015 Wiley Periodicals, Inc.

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Conditioning regimens in allo-SCT for thalassemia major

Cited by 15 publications

References 32 publications

Optimal Outcomes in Young Class 3 Patients With Thalassemia Undergoing HLA-Identical Sibling Bone Marrow Transplantation

Optimal Outcomes in Young Class 3 Patients With Thalassemia Undergoing HLA-Identical Sibling Bone Marrow Transplantation

ATG vs thiotepa with busulfan and cyclophosphamide in matched-related bone marrow transplantation for thalassemia

Thalassemia 2016: Modern medicine battles an ancient disease

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