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Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT
- Source :
- British Journal of Haematology, 171(4), 585-594, British Journal of Haematology
- Publication Year :
- 2015
-
Abstract
- We explored the feasibility of unrelated donor haematopoietic stem cell transplant (HSCT) upfront without prior immunosuppressive therapy (IST) in paediatric idiopathic severe aplastic anaemia (SAA). This cohort was then compared to matched historical controls who had undergone first-line therapy with a matched sibling/family donor (MSD) HSCT (n = 87) or IST with horse antithymocyte globulin and ciclosporin (n = 58) or second-line therapy with unrelated donor HSCT post-failed IST (n = 24). The 2-year overall survival in the upfront cohort was 96 ± 4% compared to 91 ± 3% in the MSD controls (P = 0·30) and 94 ± 3% in the IST controls (P = 0·68) and 74 ± 9% in the unrelated donor HSCT post-IST failure controls (P = 0·02).The 2-year event-free survival in the upfront cohort was 92 ± 5% compared to 87 ± 4% in MSD controls (P = 0·37), 40 ± 7% in IST controls (P = 0·0001) and 74 ± 9% in the unrelated donor HSCT post-IST failure controls (n = 24) (P = 0·02). Outcomes for upfront-unrelated donor HSCT in paediatric idiopathic SAA were similar to MSD HSCT and superior to IST and unrelated donor HSCT post-IST failure. Front-line therapy with matched unrelated donor HSCT is a novel treatment approach and could be considered as first-line therapy in selected paediatric patients who lack a MSD. © 2015 John Wiley & Sons Ltd.
- Subjects :
- Male
Pediatrics
Blood transfusion
Adenoviridae Infection
medicine.medical_treatment
Adenoviridae Infections
T-Lymphocytes
Graft vs Host Disease
Kaplan-Meier Estimate
Immunosuppressive Agent
Postoperative Complications
Retrospective Studie
hemic and lymphatic diseases
Living Donors
Young adult
Child
610 Medicine & health
Bone Marrow Transplantation
Herpesviridae Infection
Anemia, Aplastic
paediatric aplastic anaemia
Hematology
Herpesviridae Infections
aplastic anaemia
Survival Rate
Treatment Outcome
surgical procedures, operative
Child, Preschool
Histocompatibility
Cohort
Cyclosporine
Female
Case-Control Studie
Immunosuppressive Agents
Human
Adult
Living Donor
medicine.medical_specialty
Sibling
Adolescent
Disease-Free Survival
Follow-Up Studie
Young Adult
medicine
Humans
Blood Transfusion
Survival rate
Antilymphocyte Serum
Retrospective Studies
Peripheral Blood Stem Cell Transplantation
business.industry
Siblings
Case-control study
Infant
Retrospective cohort study
Length of Stay
Transplantation
T-Lymphocyte
Case-Control Studies
Quality of Life
Virus Activation
Postoperative Complication
Primary Graft Dysfunction
business
Follow-Up Studies
transplantation
Subjects
Details
- Language :
- English
- Database :
- OpenAIRE
- Journal :
- British Journal of Haematology, 171(4), 585-594, British Journal of Haematology
- Accession number :
- edsair.doi.dedup.....ddedb9e22244ae43911d589e8f768984