GuidelineHaematologica2014
Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.
Guideline in Haematologica, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 150 papers, 6 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
Bone Marrow Transplantation HLA Haploidentical After a Reduced Intensity Conditioning and Prevention of GvHD Based on Post-transplant Cyclophosphamide Administration in Patients With Severe Sickle Cell Disease
Who cites it
150 citing papers in PubMed, 6 syntheses or guidelines pooled it, 377 citations in OpenAlex.
- Conditioning regimen for hematopoietic cell transplant in transfusion dependent thalassemia: Recommendations from the EBMT practice harmonisation and guidelines committee.Bone marrow transplantation · 2026Guideline
- Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations.Bone marrow transplantation · 2025Guideline
- A Portrait of SARS-CoV-2 Infection in Patients Undergoing Hematopoietic Cell Transplantation: A Systematic Review of the Literature.Current oncology (Toronto, Ont.) · 2022Pooled it
- American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation.Blood advances · 2021Guideline
- Hematopoietic stem cell transplantation for people with β-thalassaemia.The Cochrane database of systematic reviews · 2021Pooled it
- Health-Related Quality of Life of Children and Adolescents with Sickle Cell Disease in the Middle East and North Africa Region: A systematic review.Sultan Qaboos University medical journal · 2020Pooled it
- Trial
- Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: a phase II, open-label, dose-finding study.Haematologica · 2019Trial
- Pregnancy outcome following hematopoietic cell transplantation for thalassemia major.Bone marrow transplantation · 2017Trial
- Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010.Bone marrow transplantation · 2016Trial
- Successful Management of Severe Hepatic Acute Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation in a Child With β-Thalassemia Major: Clinical Lessons From Early Therapeutic Escalation.Clinical case reports · 2026Article
- Contemporary Management of Thalassemia: A Perspective on Current Standards and the Emergence of FDA-Approved Oral Therapy.Health science reports · 2026Article
- Article
- Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative Medicine.Biomedicines · 2026Review
- Hematopoietic Cell Transplantation for Sickle Cell Disease.Children (Basel, Switzerland) · 2026Review
- Outcomes of hematopoietic stem cell transplantation for pediatric patients with transfusion-dependent thalassemia in Thailand.Clinical and experimental pediatrics · 2026Article
- Transforming the treatment of Alpha-Thalassemia: a single-center retrospective study on hematopoietic stem cell transplantation in transfusion-dependent pediatric patients.Annals of hematology · 2026Article
- FAIR data gaps and collaboration willingness among hemoglobinopathy research centers.Scientific data · 2026Article
- Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.Cell reports. Medicine · 2025Review
- Iron toxicity and hematopoietic cell transplantation: do we understand why iron affects transplant outcome?Hematology. American Society of Hematology. Education Program · 2025Review
90 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
24 authors at 17 institutions in 10 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention and treatment of complications. However, hematopoietic stem cell transplantation is still the only available curative option. The use of hematopoietic stem cell transplantation has been increasing, and outcomes today have substantially improved compared with the past three decades. Current experience world-wide is that more than 90% of patients now survive hematopoietic stem cell transplantation and disease-free survival is around 80%. However, only a few controlled trials have been reported, and decisions on patient selection for hematopoietic stem cell transplantation and transplant management remain principally dependent on data from retrospective analyses and on the clinical experience of the transplant centers. This consensus document from the European Blood and Marrow Transplantation Inborn Error Working Party and the Paediatric Diseases Working Party aims to report new data and provide consensus-based recommendations on indications for hematopoietic stem cell transplantation and transplant management.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.