Late effects after treosulfan- and busulfan-based myeloablative conditioning for allogeneic HSCT in children with malignant diseases: a comparative study

Y. V. Skvortsova; Скворцова Ю. В.; G. A. Novichkova; Новичкова Г.А.; M. A. Maschan; Масчан М.А.; D. N. Balashov; Балашов Д.Н.; L. I. Papusha; Папуша Л.И.; I. P. Shipitsina; Шипицына И.П.; L. N. Shelikhova; Шелихова Л.Н.; K. A. Voronin; Воронин К.А.; E. V. Skorobogatova; Скоробогатова Е.В.; A. A. Maschan; Масчан А.А.

doi:10.24287/1726-1708-2018-17-2-28-38

Late effects after treosulfan- and busulfan-based myeloablative conditioning for allogeneic HSCT in children with malignant diseases: a comparative study

Authors: Skvortsova Y.V.¹, Novichkova G.A.¹, Maschan M.A.¹, Balashov D.N.¹, Papusha L.I.¹, Shipitsina I.P.¹, Shelikhova L.N.¹, Voronin K.A.¹, Skorobogatova E.V.², Maschan A.A.¹
Affiliations:
1. Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology
2. ФГБУ «Российская детская клиническая больница» Минздрава России
Issue: Vol 17, No 2 (2018)
Pages: 28-38
Section: ORIGINAL ARTICLES
Submitted: 20.09.2018
Published:
URL: https://hemoncim.com/jour/article/view/173
DOI: https://doi.org/10.24287/1726-1708-2018-17-2-28-38
ID: 173

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Abstract

Treosulfan-based regimens used in conditioning for allogeneic HSCT (allo-HSCT) in children allow to reduce incidence of transplant-related mortality and early toxicity, but there are no data about long-term effects. We propose a retrospective comparative study of late complications after allo-HSCT in 135 children with hematologic malignancies, who got allo-HSCT between January 1994 and July 2011 and survived at least 1 year after HSCT. Five-year event free survival was 75.6% in the group of children who got busulfan and 66.5% in patients with treosulfan-based conditioning; 5-year nonrelapse mortality was 6% and 10.2%, respectively. Late effects analysis demonstrated that treosulfan-containing conditioning allowed to reduce the incidence of some late complications: hypergonadotropic hypogonadism cumulative incidence was 0 compared to 32.8% in busulfan-conditioning group (р = 0.012); the decrease in subclinical hypothyroidism, secondary cardiomyopathy and organic central nervous system pathology incidence was revealed. Thus treosulfan-based conditioning regimen is an effective approach in reducing of serious late complications after allogeneic HSCT when compared with data of busulfan-based conditioning long-term effects.

Keywords

late complications, allogeneic HSCT, treosulfan-based conditioning, children

About the authors

Y. V. Skvortsova

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology

Author for correspondence.
Email: yuscvo@mail.ru
ORCID iD: 0000-0002-0566-053X

MD, hematologist, Hematopoietic Stem Cell Transplantation Department

Russia 117997, Moscow, Samory Mashela st., 1

Россия

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