Decision making on HSCT in patients with hemoglobinopathies; an EBMT Pediatric Diseases Working Party and Inborn Errors Working Party scenario-based survey on physicians’ perspectives-Reference-Cited by-同舟云学术

Decision making on HSCT in patients with hemoglobinopathies; an EBMT Pediatric Diseases Working Party and Inborn Errors Working Party scenario-based survey on physicians’ perspectives

Published:2023-01-26 Issue:5 Volume:58 Page:584-586
ISSN:0268-3369
Container-title:Bone Marrow Transplantation
language:en
Short-container-title:Bone Marrow Transplant

Author:

Mekelenkamp Hilda^ORCID,de Vries Martine,Markovitch Benny,Sirait Tiarlan,Pieterse Arwen H.^ORCID,Bense Joëll^ORCID,Kleinschmidt Katharina,Albert Michael H.,Neven Bénédicte,Corbacioglu Selim^ORCID,Lankester Arjan^ORCID,Smiers Frans

Publisher

Springer Science and Business Media LLC

Subject

Transplantation,Hematology

Link

https://www.nature.com/articles/s41409-023-01926-4.pdf

Reference6 articles.

1. Leonard A, Bertaina A, Bonfim C, Cohen S, Prockop S, Purtill D, et al. Curative therapy for hemoglobinopathies: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation. Cytotherapy. 2021. https://doi.org/10.1016/j.jcyt.2021.09.003.

2. Iqbal M, Reljic T, Corbacioglu S, de la Fuente J, Gluckman E, Kumar A, et al. Systematic review/meta-analysis on efficacy of allogeneic hematopoietic cell transplantation in sickle cell disease: an international effort on behalf of the Pediatric Diseases Working Party of European Society for Blood and Marrow Transplantation and the Sickle Cell Transplantation International Consortium. Transpl Cell Ther. 2021;27:167.e1–12. https://doi.org/10.1016/j.jtct.2020.10.007.

3. Oikonomopoulou C, Goussetis E. HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe. Bone Marrow Transplant. 2021;56:2882–8. https://doi.org/10.1038/s41409-021-01461-0.

4. Krishnamurti L. Hematopoietic cell transplantation for sickle cell disease: updates and future directions. Hematol Am Soc Hematol Educ Program. 2021;2021:181–9. https://doi.org/10.1182/hematology.2021000251.

5. Veloski J, Tai S, Evans AS, Nash DB. Clinical vignette-based surveys: a tool for assessing physician practice variation. Am J Med Qual. 2005;20:151–7. https://doi.org/10.1177/1062860605274520.

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1. Pubertal attainment and Leydig cell function following pediatric hematopoietic stem cell transplantation: a three-decade longitudinal assessment;Frontiers in Endocrinology;2023-12-13

2. Hoping for a normal life: Decision‐making on hematopoietic stem cell transplantation by patients with a hemoglobinopathy and their caregivers;Pediatric Blood & Cancer;2023-12-11