The Development of New Agents for Post-Hematopoietic Stem Cell Transplantation Non-Infectious Complications in Children

Author:

Ilan Uri1ORCID,Brivio Erica1,Algeri Mattia2ORCID,Balduzzi Adriana3ORCID,Gonzalez-Vincent Marta4ORCID,Locatelli Franco2ORCID,Zwaan Christian Michel1,Baruchel Andre5,Lindemans Caroline167,Bautista Francisco1

Affiliation:

1. Princess Máxima Center for Pediatric Oncology, 3584 CS Utrecht, The Netherlands

2. Department of Hematology/Oncology and Cell and Gene Therapy, Bambino Gesù Children Hospital, 00165 Rome, Italy

3. Clinica Pediatrica Università degli Studi di Milano Bicocca, 20900 Monza, Italy

4. Department of Stem Cell Transplantation, Hospital Infantil Universitario Nino Jesus, 28009 Madrid, Spain

5. Department of Pediatric Hematology, AP-HP, Robert Debré Hospital, 75019 Paris, France

6. Division of Pediatrics, University Medical Center Utrecht, 3584 CX Utrecht, The Netherlands

7. Department of Stem Cell Transplantation, Regenerative Medicine Center, University Medical Center, 3584 CX Utrecht, The Netherlands

Abstract

Hematopoietic stem cell transplantation (HSCT) is often the only curative treatment option for patients suffering from various types of malignant diseases and some non-cancerous conditions. Nevertheless, it is associated with a high risk of complications leading to transplant-related mortality and long-term morbidity. An increasing number of therapeutic and prevention strategies have been developed over the last few years to tackle the complications arising in patients receiving an HSCT. These strategies have been mainly carried out in adults and some are now being translated into children. In this manuscript, we review the recent advancements in the development and implementation of treatment options for post-HSCT non-infectious complications in pediatric patients with leukemia and other non-malignant conditions, with a special attention on the new agents available within clinical trials. We focused on the following conditions: graft failure, prevention of relapse and early interventions after detection of minimal residual disease positivity following HSCT in acute lymphoblastic and myeloid leukemia, chronic graft versus host disease, non-infectious pulmonary complications, and complications of endothelial origin.

Publisher

MDPI AG

Subject

General Medicine

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