Page 65 - Haematologica July
P. 65
Complications in Hematology
Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience
Ferrata Storti Foundation
Ilhem Rahal,1 Claire Galambrun,1 Yves Bertrand,2 Nathalie Garnier,2 Catherine Paillard,3 Pierre Frange,4 Corinne Pondarré,5 Jean Hugues Dalle,6 Regis Peffault de Latour,7 Mauricette Michallet,8 Dominique Steschenko,9 Despina Moshous,4 Patrick Lutz,3 Jean Louis Stephan,10 Pierre Simon Rohrlich,11 Ibrahim Yakoub-Agha,12 Françoise Bernaudin,5 Christophe Piguet,13 Nathalie Aladjidi,14 Catherine Badens,15 Claire Berger,10 Gérard Socié,7 Cécile Dumesnil,16 Marie Pierre Castex,17 Marilyne Poirée,11 Anne Lambilliotte,12 Caroline Thomas,18 Pauline Simon,19 Pascal Auquier,20 Gérard Michel,1 Anderson Loundou,20 Imane Agouti15 and Isabelle Thuret,1,15
Haematologica 2018 Volume 103(7):1143-1149
1Service d’Hémato-Oncologie Pédiatrique, Hôpital d’Enfant de la Timone, Assistance Publique des Hôpitaux de Marseille; 2Service d'Hématologie et Immunologie Pédiatrique, Institut d'Hématologie et d'Oncologie Pédiatrique, Lyon; 3Service d’Hémato-Oncologie Pédiatrique, CHU de Strasbourg - Hôpital de Hautepierre; 4Service d’Immunologie Hématologie Pédiatrique, CHU Necker-Enfants Malades, Assistance Publique Hôpitaux de Paris; 5Service de Pédiatrie, Centre de Référence de la Drépanocytose, Centre Hospitalier Intercommunal de Créteil (CHIC); 6Service d’Immunologie Hématologie, Hôpital Robert Debré, Assistance Publique Hôpitaux de Paris; 7Service d’Hémato-Oncologie - Greffe, Hôpital Saint Louis, Assistance Publique Hôpitaux de Paris; 8Service d’Hématologie, Centre Hospitalier Lyon Sud, Pierre-Bénite; 9Service d’Hémato-Oncologie Pédiatrique, CHRU Nancy, Hôpitaux de Brabois, Vandœuvre-lès-Nancy; 10Service d’Immuno-Hématologie et Oncologie Pédiatrique, CHU de Saint-Étienne, Saint-Priest-en-Jarez; 11Service d’Hémato-Oncologie Pédiatrique, Hôpital l'Archet 2, CHU de Nice; 12Service de Maladies du Sang, CHRU Lille- Hôpital Claude Huriez; 13Service d’Hémato-Oncologie Pédiatrique, Hôpital de la Mère et de l’Enfant, CHU de Limoges; 14Service de Pédiatrie Médicale, Groupe Hospitalier Pellegrin Enfants, Bordeaux; 15Centre de Référence Thalassémie, Hôpital d’Enfant de la Timone, Assistance Publique des Hôpitaux Marseille; 16Service d’Immuno-Hématologie et Oncologie Pédiatrique, CHU-Hôpitaux de Rouen; 17Service d’Hémato-Oncologie Pédiatrique, Hôpital Des Enfants, CHU de Toulouse; 18Service d’Hématologie Pédiatrique, Hôpital Enfant- Adolescent, CHU Nantes; 19Service d’Hémato-Oncologie Pédiatrie, CHRU Jean Minjoz, Besançon and 20Service de Santé Publique, Assistance Publique des Hôpitaux Marseille et Université Aix-Marseille, France
ABSTRACT
In this retrospective study, we evaluate long-term complications in nearly all β-thalassemia-major patients who successfully received allo- geneic hematopoietic stem cell transplantation in France. Ninety-nine patients were analyzed with a median age of 5.9 years at transplantation. The median duration of clinical follow up was 12 years. All conditioning regimens were myeloablative, most were based on busulfan combined with cyclophosphamide, and more than 90% of patients underwent a transplant from a matched sibling donor. After transplantation, 11% of patients developed thyroid dysfunction, 5% diabetes, and 2% heart fail- ure. Hypogonadism was present in 56% of females and 14% of males. Female patients who went on to normal puberty after transplant were significantly younger at transplantation than those who experienced delayed puberty (median age 2.5 vs. 8.7 years). Fertility was preserved in 9 of 27 females aged 20 years or older and 2 other patients became preg- nant following oocyte donation. In addition to patient’s age and higher serum ferritin levels at transplantation, time elapsed since transplant was significantly associated with decreased height growth in multivariate analysis. Weight growth increased after transplantation particularly in females, 36% of adults being overweight at last evaluation. A compre- hensive long-term monitoring, especially of endocrine late effects, is required after hematopoietic stem cell transplantation for thalassemia.
Correspondence:
isabelle.thuret@ap-hm.fr
Received: November 9, 2017. Accepted: March 23, 2018. Pre-published: March 29, 2018.
doi:10.3324/haematol.2017.183467
Check the online version for the most updated information on this article, online supplements, and information on authorship & disclosures: www.haematologica.org/content/103/7/1143
©2018 Ferrata Storti Foundation
Material published in Haematologica is covered by copyright. All rights are reserved to the Ferrata Storti Foundation. Use of published material is allowed under the following terms and conditions: https://creativecommons.org/licenses/by-nc/4.0/legalcode. Copies of published material are allowed for personal or inter- nal use. Sharing published material for non-commercial pur- poses is subject to the following conditions: https://creativecommons.org/licenses/by-nc/4.0/legalcode, sect. 3. Reproducing and sharing published material for com- mercial purposes is not allowed without permission in writing from the publisher.
haematologica | 2018; 103(7)
1143
ARTICLE
