Risk factors affecting outcome of unrelated cord blood transplantation for children with familial haemophagocytic lymphohistiocytosis
Eurocord/ Monacord, Cord Blood Committee of Cellular Therapy, Immunobiology and Inborn Errors Working Parties of the European Blood and Marrow Transplant Group
(2019) British Journal of Haematology, volume 184, issue 3, pp. 397 - 404
(Article)
Abstract
Allogeneic haematopoietic stem cell transplantation is still the only available curative option for Familial Haemophagocytic Lymphohistiocytosis (FHLH). Most studies report outcomes after bone marrow or peripheral blood stem cell transplantation. We analysed the outcomes of 118 children with FHLH undergoing single-unit umbilical cord blood transplantation performed from 1996 to 2014.
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Myeloablative conditioning regimen was given to 90% of the patients, and was mostly busulfan-based (n = 81, 76%), including anti-thymocyte globulin or alemtuzumab (n = 102, 86%). The cumulative incidence of Day 60 neutrophil engraftment was 85%; and that of non-relapse mortality and acute graft-versus-host disease (GvHD) was 21% and 33% at 100 days, respectively. The 6-year cumulative incidence of chronic GvHD was 17% and the 6-year probability of overall survival was 55%. In multivariate analysis, children receiving a graft with a total nucleated cell dose greater than 9·9 × 107/kg had a better overall survival (hazard ratio [HR]: 0·49, 95% CI: 0·27–0·88, P = 0·02). Degree of human leucocyte antigen (HLA) matching was associated with improved disease-free survival (5/6 vs. 6/6 HR: 2·11, 95% confidence interval [CI]: 1·01–4·4, P = 0·05 and ≤4/6 vs. 6/6, HR: 2·82, CI: 1·27–6·23, P = 0·01). Umbilical cord blood transplantation with a high cell dose and good HLA match is a suitable alternative option to haematopoietic stem cell transplantation in children with FHLH who lack a HLA-matched donor.
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Keywords: Familial haemophagocytic lymphohistiocytosis, Paediatric, transplant outcomes, Umbilical cord blood transplantation, unrelated donor, Hematology
ISSN: 0007-1048
Publisher: Wiley-Blackwell
Note: Funding Information: 1Bone Marrow Transplant Department, Great Ormond Street Hospital NHS Trust, London, UK, 2Eurocord, Hôpital Saint Louis, Paris, France, 3Monacord, Centre Scientifique de Monaco, Monaco, Monaco, 4Department of Paediatric Haematology and Oncology, Instituto di Ricovero e Cura a Carattere Scientifico (IRCCS) “Bambino Gesu” Children’s Hospital, Rome, 5Department of Paediatric Sciences, University of Pavia, Pavia, Italy, 6Paediatric Blood and Marrow Transplantation Programme, Paediatric Blood and Marrow Transplantation Programme University Hospital Utrecht, Utrecht, The Netherlands, 7Department of Paediatric Haematology and Oncology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia, 8Paediatric Haematology Oncology, Fon-dazione IRCCS Policlinico San Matteo, Pavia, Italy, 9Department of Paediatric Haematology and Oncology, Hopital d‘Enfants de la Timone, CHU, Marseille, France, 10Department of Paediatric Haematology and Oncology, University Hospital Motol, Prague, Czech Republic, 11Department of Paediatrics, Leiden University Medical Centre, Leiden, The Netherlands, 12Haematology, Transfusion and Cell therapy service, Universidade de São Paulo, São Paulo, Brazil and 13Haematology Unit, Churchill Hospital, University of Oxford, Oxford, UK Funding Information: The authors thank the collaborating transplant centres for sharing patient information: Saudi Arabia – Riyadh – King Faisal Specialist Hospital and Research Centre; The Netherlands – Utrecht – University Medical Centre; Italy – Pavia – Fon dazione IRCCS Policlinico San Matteo; United Kingdom – London – Great Ormond Street Hospital; The Netherlands – Leiden – University Medical Centre; France – Marseille – La Timone (pédiatrie); Czech Republic – Prague – University Hospital Motol; Belgium – Leuven – University Hospital Gasthuisberg Dept. of Haematology; United Kingdom – Sheffield – Royal Hallamshire Hospital; Italy – Genova – Institute G. Gaslini; Turkey – Antalya – Akdeniz University Medical School; Spain – Madrid – Niño Jesus Children‘s Hospital; Sweden – Uppsala – University Hospital; Sweden – Goeteborg – Sahlgrenska University Hospital; Italy – Torino – Ospedale Infantile Regina Margherita Onco-Ematologia Pediatrica; Spain – Barcelona – Hospital Vall d‘Hebron; United Kingdom – Manchester – Department of Paediatric Haematology; Austria – Graz – Medical University; Turkey – Izmir – Ege University; Russia – Moscow – Russian Children’s Hospital; Ireland – Dublin – Our Lady’s Hospital for Sick Children; France – Lyon – Debrousse (pédiatrie); France – Strasbourg – Hautepierre-pédiatrie; Sweden – Stockholm – Karolinska University Hospital Children‘s Hospital; Norway – Rikshospi-talet Department of Medicine – Paediatrics; Spain – Barcelona – Hospital Santa Creu i Sant Pau; United Kingdom – Newcastle-Upon-Tyne – Newcastle General Hospital Dept. of Paediatric Immunology; Italy – Monza Ospedale San Gerardo Clinica Pediatrica dell’Universita di Milano Bicocca; Portugal – Porto – Inst. Portugues de Oncologia do Porto; Switzerland – Zu€rich – University Children’s Hospital; Canada – Montréal – Ste-Justine Hospital; Israel – Tel-Hashomer – Edmond & Lily Safra Children‘s Hospital, Chaim Sheba Med Centre; Germany – Hamburg – University Hospital Eppendorf; Italy – Verona – Poli-clinico G.B. Rossi; Iran – Teheran – Shariati Hospital; France – Strasbourg – Hautepierre (adulte); Australia Randwick Sydney Children’s Hospital; Italy – Brescia – Universitàdegli Studi di Brescia; Israel – Petach-Tikva – Schneider Children’s Medical Centre of Israel; Spain – Sevilla – Hospital Universitario Virgen del Rocío; New Zealand – Christchurch – Canterbury Health Laboratories; Turkey – Antalya – Medical Park Hospitals; Turkey – Istanbul – Acibadem University Atakent Hospital Paediatric BMT Unit; Turkey – Istanbul – Medical Park Goztepe Hospital Paediatric Stem Cell Transplantation Unit. Publisher Copyright: © 2018 British Society for Haematology and John Wiley & Sons Ltd
(Peer reviewed)