Trasplante de médula ósea en pacientes con anemia falciforme. Experiencia en un centro

Morin, M; Cela, Elena; Garrido, Carmen; Cancho, Eduardo Bardón; Hoyo, Alejandra Aguado del; Pascual, Cristina; Pérez‐Corral, Ana; Beléndez, Cristina

doi:10.1016/j.anpedi.2016.03.014

Cited by 6 publications

(3 citation statements)

References 22 publications

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“…Of these 11 patients, one died from acute GVHD of the gut, one had secondary graft failure with reoccurrence of SCD symptoms, and one had stable mixed chimerism. [14] This cohort had a particularly good rate of engraftment, although it’s not exactly clear why their outcomes were better since they used standard conditioning and GVHD prophylaxis regimens. Further analysis of this cohort should be undertaken to determine factors that contributed to high rates of engraftment.…”

Section: Hla-matched Sibling Transplantsmentioning

confidence: 99%

Allogeneic stem cell transplantation for sickle cell disease

Robinson

Fuchs

2016

Current Opinion in Hematology

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Purpose of Review As the safety and availability of allogeneic hematopoietic stem cell transplantation (HSCT) have improved, this procedure is becoming a viable option for non-malignant conditions such as sickle cell disease (SCD). There are very few treatment options available for SCD, and even with optimal care SCD patients still suffer from a myriad of comorbidities to multiple organ systems and have a decreased life span. In this review we will summarize results from trials of HSCT for children or adults with SCD using a variety of graft sources as well as conditioning and GVHD prophylaxis regimens, and discuss the unique challenges that arise in these patients. Recent Findings AlloHSCT for SCD has been performed on small numbers of patients at multiple centers around the world using several different transplant platforms, and early outcomes are encouraging. Overall survival is excellent, although graft failure remains a challenge. Summary As alloHSCT becomes safer and more widely available, the procedure should be considered for patients with severe disease phenotypes in whom the potential benefits of transplantation outweigh the complications from the disease. AlloHSCT has been shown to reverse or at least halt the progression of end-organ damage secondary to SCD. More research is needed to understand the mechanisms underlying graft failure in SCD recipients, as well as to understand the biopsychosocial underpinnings of persistent pain in the post-transplant period to maximize the benefit from the transplant procedure.

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Section: Hla-matched Sibling Transplantsmentioning

confidence: 99%

Allogeneic stem cell transplantation for sickle cell disease

Robinson

Fuchs

2016

Current Opinion in Hematology

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“…While therapies directed at repairing the genetic defects are being developed, 16 still hematopoietic stem cell transplantation (HSCT) is currently the most used curative option for SCD 17‐23 . However, only a selected group of patients have been able to benefit of this procedure due to donor availability, socioeconomic reasons, and transplant‐related complications such as transplant‐related mortality, graft failure (GF), acute and chronic graft‐versus‐host disease (GvHD), and infertility 24,25 .…”

Section: Introductionmentioning

confidence: 99%

“…[13][14][15] While therapies directed at repairing the genetic defects are being developed, 16 still hematopoietic stem cell transplantation (HSCT) is currently the most used curative option for SCD. [17][18][19][20][21][22][23] However, only a selected group of patients have been able to benefit of this procedure due to donor availability, socioeconomic reasons, and transplant-related complications such as transplant-related mortality, graft failure (GF), acute and chronic graftversus-host disease (GvHD), and infertility. 24,25 An international survey from the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) and the Center for International Blood and Marrow Transplantation (CIBMTR) showed an overall survival (OS) of 91.4% after matched sibling donor (MSD) HSCT.…”

Section: Introductionmentioning

confidence: 99%

Matched sibling donor stem cell transplantation for sickle cell disease: Results from the Spanish group for bone marrow transplantation in children

Benítez‐Carabante

Beléndez

González‐Vicent

et al. 2021

European J of Haematology

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Objectives The prevalence of sickle cell disease (SCD) in Spain is markedly inferior compared with other European and Mediterranean countries. However, the diagnosis of new patients with SCD is expected to increase. In this multicenter retrospective study, we analyze the hematopoietic stem cell transplantation (HSCT) results obtained in Spain. Methods Forty‐five patients who underwent a matched sibling donor (MSD) HSCT between 1999 and 2018 were included. Primary endpoint was event‐free survival (EFS), and secondary endpoints included acute and chronic graft‐versus‐host disease (GvHD) and overall survival (OS). Results Bone marrow was the most frequent stem cell source (93.3%). Most patients received a conditioning regimen based on busulfan and cyclophosphamide (69%). Cumulative incidence of grade III‐IV acute GvHD and chronic GvHD was 6.8% (95% CI: 2.3%‐20.1%) and 5.4% (95% CI: 1.38%‐19.9%), respectively. EFS and overall survival (OS) at 3 years post‐HSCT were 89.4% (95% CI: 73.9%‐95.9%) and 92.1% (95% CI: 77.2%‐97.4%), respectively. All patients aged ≤ 5 presented 100% EFS and OS. Conclusions An early referral to HSCT centers should be proposed early in life, before severe complications occur. MSD HSCT should be considered a curative option for all patients aged ≤ 5 years and for older pediatric patients who present complications derived from the disease.

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Unique Challenges of Hematopoietic Stem Cell Transplant for Sickle Cell Disease

Khandelwal

Grimley

2017

Sickle Cell Disease and Hematopoietic Stem Cell Transplantation

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Trasplante de médula ósea en pacientes con anemia falciforme. Experiencia en un centro

Cited by 6 publications

References 22 publications

Allogeneic stem cell transplantation for sickle cell disease

Allogeneic stem cell transplantation for sickle cell disease

Matched sibling donor stem cell transplantation for sickle cell disease: Results from the Spanish group for bone marrow transplantation in children

Unique Challenges of Hematopoietic Stem Cell Transplant for Sickle Cell Disease

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