Hematopoietic stem cell transplantation in pediatric patients with β-thalassemia and sickle cell disease: An experience of the Spanish Working Group for Bone Marrow Transplantation in Children (GETMON)

Alonso, Laura; González‐Vicent, Marta; Beléndez, Cristina; Badell, Isabel; Sastre, Ana; Rodríguez-Villa, Antonia; Bermúdez-Cortés, Mar; Hladun, Raquel; Heredia, Cristina Díaz de

doi:10.1016/j.medcle.2018.12.010

Cited by 5 publications

(6 citation statements)

References 23 publications

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“…Allogeneic haematopoietic stem cell transplantation (Allo-HSCT) is currently the only potential cure for the disease, and previous studies have shown that Allo-HSCT significantly improves the quality of life (QOL) of children in developing regions [5,6]. However, Allo-HSCT is currently difficult to popularize clinically due to limited marrow sources and its high cost [7]. The possible sequelae of Allo-HSCT, such as chronic graft-versus-host disease and subsequent cancers, also cannot be ignored [8,9].…”

Section: Introductionmentioning

confidence: 99%

Psychological intervention in children with transfusion‐dependent β‐thalassaemia

Wang

Huang

Hong³

2021

Vox Sanguinis

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Background and Objectives: Transfusion-dependent β-thalassaemia can lead to severe psychological issues in paediatric and adolescent patients. However, the psychological interventions for these patients are limited in clinical practice. We aimed to investigate the impact of a 3-month psychological intervention on the quality of life (QOL) of children with β-thalassaemia (12-18 years old) who relied on blood transfusion in this study. Materials and Methods:In the current randomized controlled trial, a total of 143 paediatric or adolescent patients (12-18 years old) with transfusion-dependent β-thalassaemia were recruited. They were randomized into the control group (n = 71) who received standard physiological treatment and the intervention group (n = 72) who received a 3-month intervention in addition to standard physiological treatment. The effects of the interventions on the QOL and psychological outcomes of these participants were analysed. Results:The 3-month intervention significantly improved the scores of PedsQoL 4.0 Generic Core Scales of paediatric patients with transfusion-dependent β-thalassaemia. It also significantly improved the psychological status and alleviated the depression among children and adolescent patients by alleviating anhedonia, negative mood and negative self-esteem among them. Conclusion:Psychological intervention has positive effects on the treatment for children with transfusion-dependent β-thalassaemia.

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Section: Introductionmentioning

confidence: 99%

Psychological intervention in children with transfusion‐dependent β‐thalassaemia

Wang

Huang

Hong³

2021

Vox Sanguinis

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“…The unrelated cord blood stem cell transplant and haploidentical stem cell have been found to be less successful due to an increase in graft vs. host disease. Bone marrow transplantation has helped in improving the quality of life of the patient by eliminating the repeated hospital admission [2].…”

Section: Editorialmentioning

confidence: 99%

Allogeneic Bone Marrow Transplantation from Haplomatched Sibling Donor in a Pediatric Patient with Sickle Cell Disease with Multiple Painful Crises: An Experience

Rai¹,

Mishra²,

Jain³

et al. 2022

annhematoloncol

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Bone marrow transplantation is the only cure for sickle cell disease but this curative therapy unfortunately lead to complication and several times death. Bone marrow transplantation indicated only for sickle cell patients with severe sickle cell disease who have complications including strokes, acute chest syndrome, recurrent pain crisis and exchange transfusions, nephropathy retinopathy, osteonecrosis of multiple joints and priapism. All sickle cell patients are not eligible for bone marrow transplantation due to associated toxicity [1]. Transplant is performed when benefit outweighs the risk. There has been much development and improvement in the bone marrow transplant since the first transplant done in 1984. This technique includes using chemotherapy for conditioning to remove the recipients cells and replacing it with donor cells free of sickling. Main problem limiting the transplant to all patients include getting a matched donor, risk associated with the procedure; including graft vs. host disease and cost. Hematopoietic stem cell transplant is safest when a matched donor is available. Even with this, there is still risk of graft rejection and chronic graft verses host disease. The unrelated cord blood stem cell transplant and haploidentical stem cell have been found to be less successful due to an increase in graft vs. host disease. Bone marrow transplantation has helped in improving the quality of life of the patient by eliminating the repeated hospital admission [2].

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“…Proof-of-concept therapies for b-thalassemia Hematopoietic stem cell transplantation (HSCT) remains the definitive cure currently available for patients with b-thalassemia major. [40] However, the limits of suitable donors and costs minimize their clinical application. It is accompanied by potential immune-mediated rejection and graft-versus-host disease (GVHD).…”

Section: Update On the Genetic Control Of Fetal Hemoglobinmentioning

confidence: 99%

Molecular genetics of β-thalassemia

et al. 2021

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β-thalassemia is a hereditary hematological disease caused by over 350 mutations in the β-globin gene (HBB). Identifying the genetic variants affecting fetal hemoglobin (HbF) production combined with the α-globin genotype provides some prediction of disease severity for β-thalassemia. However, the generation of an additive composite genetic risk score predicts prognosis, and guide management requires a larger panel of genetic modifiers yet to be discovered.Presently, using data from prior clinical trials guides the design of further research and academic studies based on gene augmentation, while fundamental insights into globin switching and new technology developments have inspired the investigation of novel gene therapy approaches.Genetic studies have successfully characterized the causal variants and pathways involved in HbF regulation, providing novel therapeutic targets for HbF reactivation. In addition to these HBB mutation-independent strategies involving HbF synthesis de-repression, the expanding genome editing toolkit provides increased accuracy to HBB mutation-specific strategies encompassing adult hemoglobin restoration for personalized treatment of hemoglobinopathies. Allogeneic hematopoietic stem cell transplantation was, until very recently, the curative option available for patients with transfusion-dependent β-thalassemia. Gene therapy currently represents a novel therapeutic promise after many years of extensive preclinical research to optimize gene transfer protocols.We summarize the current state of developments in the molecular genetics of β-thalassemia over the last decade, including the mechanisms associated with ineffective erythropoiesis, which have also provided valid therapeutic targets, some of which have been shown as a proof-of-concept.

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Hematopoietic stem cell transplantation in pediatric patients with β-thalassemia and sickle cell disease: An experience of the Spanish Working Group for Bone Marrow Transplantation in Children (GETMON)

Cited by 5 publications

References 23 publications

Psychological intervention in children with transfusion‐dependent β‐thalassaemia

Psychological intervention in children with transfusion‐dependent β‐thalassaemia

Allogeneic Bone Marrow Transplantation from Haplomatched Sibling Donor in a Pediatric Patient with Sickle Cell Disease with Multiple Painful Crises: An Experience

Molecular genetics of β-thalassemia

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