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

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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.

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

Russian Federation

G. A. Novichkova

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

ORCID iD: 0000-0002-2322-5734
Russian Federation

M. A. Maschan

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

ORCID iD: 0000-0003-1735-0093
Russian Federation

D. N. Balashov

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

ORCID iD: 0000-0003-2689-0569
Russian Federation

L. I. Papusha

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

ORCID iD: 0000-0001-7750-5216
Russian Federation

I. P. Shipitsina

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

ORCID iD: 0000-0001-8687-5497
Russian Federation

L. N. Shelikhova

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

ORCID iD: 0000-0003-0520-5630
Russian Federation

K. A. Voronin

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

ORCID iD: 0000-0001-7578-9657
Russian Federation

E. V. Skorobogatova

ФГБУ «Российская детская клиническая больница» Минздрава России

ORCID iD: 0000-0003-4431-1444
Russian Federation

A. A. Maschan

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

ORCID iD: 0000-0002-0016-6698
Russian Federation

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Copyright (c) 1970 Skvortsova Y.V., Novichkova G.A., Maschan M.A., Balashov D.N., Papusha L.I., Shipitsina I.P., Shelikhova L.N., Voronin K.A., Skorobogatova E.V., Maschan A.A.

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