Risk factors affecting outcome of unrelated cord blood transplantation for children with familial haemophagocytic lymphohistiocytosis.

Autor: Furtado-Silva JM; Bone Marrow Transplant Department, Great Ormond Street Hospital NHS Trust, London, UK., Paviglianiti A; Eurocord, Hôpital Saint Louis, Paris, France.; Monacord, Centre Scientifique de Monaco, Monaco, Monaco., Ruggeri A; Eurocord, Hôpital Saint Louis, Paris, France.; Monacord, Centre Scientifique de Monaco, Monaco, Monaco.; Department of Paediatric Haematology and Oncology, Instituto di Ricovero e Cura a Carattere Scientifico (IRCCS) 'Bambino Gesù' Children's Hospital, Rome, Italy.; Department of Paediatric Sciences, University of Pavia, Pavia, Italy., Boelens JJ; Paediatric Blood and Marrow Transplantation Programme, Paediatric Blood and Marrow Transplantation Programme University Hospital Utrecht, Utrecht, The Netherlands., Veys P; Bone Marrow Transplant Department, Great Ormond Street Hospital NHS Trust, London, UK., Ahmari AA; Department of Paediatric Haematology and Oncology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia., Zecca M; Paediatric Haematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy., Locatelli F; Department of Paediatric Haematology and Oncology, Instituto di Ricovero e Cura a Carattere Scientifico (IRCCS) 'Bambino Gesù' Children's Hospital, Rome, Italy.; Department of Paediatric Sciences, University of Pavia, Pavia, Italy., Michel G; Department of Paediatric Haematology and Oncology, Hopital d'Enfants de la Timone, CHU, Marseille, France., Volt F; Eurocord, Hôpital Saint Louis, Paris, France.; Monacord, Centre Scientifique de Monaco, Monaco, Monaco., Kenzey C; Eurocord, Hôpital Saint Louis, Paris, France.; Monacord, Centre Scientifique de Monaco, Monaco, Monaco., Sedlacek P; Department of Paediatric Haematology and Oncology, University Hospital Motol, Prague, Czech Republic., Rao K; Bone Marrow Transplant Department, Great Ormond Street Hospital NHS Trust, London, UK., Lankester A; Department of Paediatrics, Leiden University Medical Centre, Leiden, The Netherlands., Gluckman E; Eurocord, Hôpital Saint Louis, Paris, France.; Monacord, Centre Scientifique de Monaco, Monaco, Monaco., Rocha V; Eurocord, Hôpital Saint Louis, Paris, France.; Monacord, Centre Scientifique de Monaco, Monaco, Monaco.; Haematology, Transfusion and Cell therapy service, Universidade de São Paulo, São Paulo, Brazil.; Haematology Unit, Churchill Hospital, University of Oxford, Oxford, UK.
Jazyk: angličtina
Zdroj: British journal of haematology [Br J Haematol] 2019 Feb; Vol. 184 (3), pp. 397-404. Date of Electronic Publication: 2018 Nov 21.
DOI: 10.1111/bjh.15642
Abstrakt: 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. 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 × 10 7 /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.
(© 2018 British Society for Haematology and John Wiley & Sons Ltd.)
Databáze: MEDLINE
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