Family cord blood banking for sickle cell disease: a twenty-year experience in two dedicated public cord blood banks

Rafii, Hanadi; Bernaudin, Françoise; Rouard, Hélène; Ruggeri, Annalisa; Cavazzana, Marina; Gauthereau, Valérie; Stanislas, Aurélie; Benkerrou, Malika; Montalembert, Mariane de; Ferry, C; Girot, Robert; Kamdem, Annie; Gour, Joelle; Touboul, C.; Cras, Audrey; Kuentz, Mathieu; Rieux, Claire; Volt, Fernanda; Cappelli, Barbara; Maio, Karina Tozatto; Paviglianiti, Annalisa; Kenzey, Chantal; Larghero, Jérôme; Gluckman, Éliane

doi:10.3324/haematol.2016.163055

Cited by 9 publications

(2 citation statements)

References 39 publications

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“…Additionally, it is to balance the differences in efficacy with the risk of GVHD, because these trials were not randomized. Despite the nonsignificance in OS, 14,91,[92][93][94][95][96] it is important to note that no deaths were observed after HSCT with UCB.…”

Section: Recommendationmentioning

confidence: 99%

American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation

et al. 2021

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Background: Sickle cell disease (SCD) is a life-limiting inherited hemoglobinopathy that results in significant complications and affects quality of life. Hematopoietic stem cell transplantation (HSCT) is currently the only curative intervention for SCD; however, guidelines are needed to inform how to apply HSCT in clinical practice. Objective: These evidence-based guidelines of the American Society of Hematology (ASH) are intended to support patients, clinicians, and health professionals in their decisions about HSCT for SCD. Methods: The multidisciplinary guideline panel formed by ASH included 2 patient representatives and was balanced to minimize potential bias from conflicts of interest. The Mayo Evidence-Based Practice Research Program supported the guideline development process, including performing systematic evidence reviews (through 2019). The panel prioritized clinical questions and outcomes according to their importance for clinicians and patients. The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including GRADE Evidence-to-Decision frameworks, to assess evidence and make recommendations, which were subject to public comment. Results: The panel agreed on 8 recommendations to help patients and providers assess how individuals with SCD should consider the timing and type of HSCT. Conclusions: The evidence review yielded no randomized controlled clinical trials for HSCT in SCD; therefore, all recommendations are based on very low certainty in the evidence. Key recommendations include considering HSCT for those with neurologic injury or recurrent acute chest syndrome at an early age and to improve nonmyeloablative regimens. Future research should include the development of a robust SCD registry to serve as a comparator for HSCT studies.

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Section: Recommendationmentioning

confidence: 99%

American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation

et al. 2021

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“…The [76][77][78], malaria [79,80] and HIV [81,82]. [91][92][93][94], cord blood serum [95], as well as the whole cord blood [96].…”

Section: Cryopreservation In Human Reproductive Medicinementioning

confidence: 99%

Translation of Cryobiological Techniques to Socially Economically Deprived Populations—Part 1: Cryogenic Preservation Strategies

Buriak

Fleck

Goltsev

et al. 2020

Journal of Medical Devices

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Use of cold for preservation of biological materials, avoidance of food spoilage and to manage a variety of medical conditions has been known for centuries. The cryobiological science justified these applications in the 1960s increasing their use in expanding global activities. However, the engineering and technological aspects associated with cryobiology can be expensive and this raises questions about the abilities of resource-restricted low and middle income countries (LMICs) to benefit from the advances. This review was undertaken to understand where or how access to cryobiological advances currently exist and the constraints on their usage. The subject areas investigated were based on themes which commonly appear in the journal Cryobiology. This led in the final analysis for separating the review into two parts, with the first part dealing with cold applied for biopreservation of living cells and tissues in science, health care and agriculture, and the second part dealing with cold destruction of tissues in medicine. The limitations of the approaches used are recognized, but as a first attempt to address these topics surrounding access to cryobiology in LMICs, the review should pave the way for future more subject-specific assessments of the true global uptake of the benefits of cryobiology.

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Hemoglobinopathies (Sickle Cell Disease and Thalassemia)

Cappelli,

Gluckman,

Corbacioglu

et al. 2024

The EBMT Handbook

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Hematopoietic cell transplantation (HCT) using an HLA-matched sibling donor is a well-established curative therapy for pediatric patients with sickle cell disease (SCD) and transfusion-dependent thalassemias (TDT). In order to expand the donor pool, new approaches such as related haploidentical donor HCT have been used with encouraging results. These approaches aim for a higher overall survival, an effective reduction of acute and chronic GvHD and a reduced toxicity. Due to these alternative approaches and adult patients being increasingly transplanted, the number of HCT has dramatically increased in the last decade. Furthermore, different gene therapy and gene editing strategies are being developed in clinical trials, showing promising results.

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Family cord blood banking for sickle cell disease: a twenty-year experience in two dedicated public cord blood banks

Cited by 9 publications

References 39 publications

American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation

American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation

Translation of Cryobiological Techniques to Socially Economically Deprived Populations—Part 1: Cryogenic Preservation Strategies

Hemoglobinopathies (Sickle Cell Disease and Thalassemia)

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