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

Research output: Contribution to journalArticle

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. 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.

Original languageEnglish
Number of pages8
JournalBritish Journal of Haematology
DOIs
Publication statusE-pub ahead of print - Nov 21 2018

Fingerprint

Hemophagocytic Lymphohistiocytosis
HLA Antigens
Fetal Blood
Transplantation
Hematopoietic Stem Cell Transplantation
Graft vs Host Disease
Confidence Intervals
Peripheral Blood Stem Cell Transplantation
Busulfan
Antilymphocyte Serum
Survival
Incidence
Disease-Free Survival
Neutrophils
Multivariate Analysis
Bone Marrow
Outcome Assessment (Health Care)
Tissue Donors
Transplants
Mortality

Cite this

Eurocord/ Monacord, Cord Blood Committee of Cellular Therapy, Immunobiology and Inborn Errors Working Parties of the European Blood and Marrow Transplant Group (2018). Risk factors affecting outcome of unrelated cord blood transplantation for children with familial haemophagocytic lymphohistiocytosis. British Journal of Haematology. https://doi.org/10.1111/bjh.15642

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.

In: British Journal of Haematology, 21.11.2018.

Research output: Contribution to journalArticle

Eurocord/ Monacord, Cord Blood Committee of Cellular Therapy, Immunobiology and Inborn Errors Working Parties of the European Blood and Marrow Transplant Group 2018, 'Risk factors affecting outcome of unrelated cord blood transplantation for children with familial haemophagocytic lymphohistiocytosis', British Journal of Haematology. https://doi.org/10.1111/bjh.15642
Eurocord/ Monacord, Cord Blood Committee of Cellular Therapy, Immunobiology and Inborn Errors Working Parties of the European Blood and Marrow Transplant Group. Risk factors affecting outcome of unrelated cord blood transplantation for children with familial haemophagocytic lymphohistiocytosis. British Journal of Haematology. 2018 Nov 21. https://doi.org/10.1111/bjh.15642
Eurocord/ Monacord, Cord Blood Committee of Cellular Therapy, Immunobiology and Inborn Errors Working Parties of the European Blood and Marrow Transplant Group. / Risk factors affecting outcome of unrelated cord blood transplantation for children with familial haemophagocytic lymphohistiocytosis. In: British Journal of Haematology. 2018.
@article{63fe452be52f4f9e9243bafaebe4519e,
title = "Risk factors affecting outcome of unrelated cord blood transplantation for children with familial haemophagocytic lymphohistiocytosis",
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. 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.",
author = "{Eurocord/ Monacord, Cord Blood Committee of Cellular Therapy, Immunobiology and Inborn Errors Working Parties of the European Blood and Marrow Transplant Group} and Furtado-Silva, {Juliana Montibeller} and Annalisa Paviglianiti and Annalisa Ruggeri and Boelens, {Jaap Jan} and Paul Veys and Ahmari, {Ali Abdallah} and Marco Zecca and Franco Locatelli and Gerard Michel and Fernanda Volt and Chantal Kenzey and Petr Sedlacek and Kanchan Rao and Arjan Lankester and Eliane Gluckman and Vanderson Rocha",
note = "{\circledC} 2018 British Society for Haematology and John Wiley & Sons Ltd.",
year = "2018",
month = "11",
day = "21",
doi = "10.1111/bjh.15642",
language = "English",
journal = "British Journal of Haematology",
issn = "0007-1048",
publisher = "John Wiley & Sons, Ltd (10.1111)",

}

TY - JOUR

T1 - Risk factors affecting outcome of unrelated cord blood transplantation for children with familial haemophagocytic lymphohistiocytosis

AU - Eurocord/ Monacord, Cord Blood Committee of Cellular Therapy, Immunobiology and Inborn Errors Working Parties of the European Blood and Marrow Transplant Group

AU - Furtado-Silva, Juliana Montibeller

AU - Paviglianiti, Annalisa

AU - Ruggeri, Annalisa

AU - Boelens, Jaap Jan

AU - Veys, Paul

AU - Ahmari, Ali Abdallah

AU - Zecca, Marco

AU - Locatelli, Franco

AU - Michel, Gerard

AU - Volt, Fernanda

AU - Kenzey, Chantal

AU - Sedlacek, Petr

AU - Rao, Kanchan

AU - Lankester, Arjan

AU - Gluckman, Eliane

AU - Rocha, Vanderson

N1 - © 2018 British Society for Haematology and John Wiley & Sons Ltd.

PY - 2018/11/21

Y1 - 2018/11/21

N2 - 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 × 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.

AB - 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 × 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.

U2 - 10.1111/bjh.15642

DO - 10.1111/bjh.15642

M3 - Article

C2 - 30460979

JO - British Journal of Haematology

JF - British Journal of Haematology

SN - 0007-1048

ER -