TY - JOUR
T1 - Late Effects in Pediatric Allogeneic Hematopoietic Stem Cell Transplantation for Nonmalignant Diseases
T2 - Proxy- and Patient-Reported Outcomes
AU - Bense, Jo?ll E.
AU - Haverman, Lotte
AU - von Asmuth, Erik G. J.
AU - Louwerens, Marloes
AU - Luijten, Michiel A. J.
AU - Stiggelbout, Anne M.
AU - Lankester, Arjan C.
AU - de Pagter, Anne P. J.
N1 - Funding Information: The authors thank all the participants for their time and for sharing their personal experiences after this high-intensity treatment. Financial disclosure: The authors declare that no funds, grants, or other support were received during the preparation of this manuscript. Conflict of interest statement: There are no conflicts of interest to report. Authorship statement: J.B. collected data, analyzed results, and drafted the manuscript. J.B. and E.A. performed the statistical analysis. MLu. provided expert opinion on statistical analysis. J.B. L.H. M.Lo. A.S. A.L. A.P. interpreted the data. A.P. supervised the project. All authors critically revised the manuscript, approved the final version for publication, and are accountable for this work. Data availability: The datasets generated during and/or analyzed during this study are available from the corresponding author on reasonable request. Financial disclosure: See Acknowledgments on page 186.e9. Publisher Copyright: © 2022 The American Society for Transplantation and Cellular Therapy
PY - 2023/3
Y1 - 2023/3
N2 - Survival rates in pediatric hematopoietic stem cell transplantation (HSCT) for nonmalignant diseases have improved due to advances in conditioning regimens, donor selection, and prophylaxis and treatment of infections and graft-versus-host disease. Insight into the long-term patient-reported outcomes (PROs) after pediatric HSCT for nonmalignant disease is lacking but essential for optimal shared decision making, counseling, and quality of care. The purpose of this research was to determine long-term patient-reported outcomes in allogeneic pediatric HSCT for nonmalignant diseases and to compare these results with Dutch reference data. This single-center cohort study evaluated PROs (PedsQL 4.0, PROMIS item banks), self- or proxy-reported, among patients at ?2 years after pediatric allogeneic HSCT for nonmalignant disease. Mean scores were compared with those of the Dutch general population. Of 171 eligible patients, 119 participated, for a 70% response rate. The median patient age was 15.8 years (range, 2 to 49 years), and the median duration of follow-up was 8.7 years (range, 2 to 34 years). Indications for HSCT included inborn errors of immunity (n = 41), hemoglobinopathies (n = 37), and bone marrow failure (n = 41). Compared with reference data, significantly lower scores were found in adolescents (age 13 to 17 years) on the Total, Physical Health, and School Functioning PedsQL subscales. Significantly more Sleep Disturbance was reported in children (age 8 to 18 years). On the other hand, significantly better scores were seen on PROMIS Fatigue (age 5 to 7 years) and Pain Interference (age 8 to 18 years) and, in adults (age 19 to 30 years), on Depressive Symptoms and Sleep Disturbance. This study showed better or comparable very long-term PROs in patients after pediatric HSCT for nonmalignant diseases compared with the reference population. Children and adolescents seem to be the most affected, indicating the need for supportive care to prevent impaired quality of life and, more importantly, to amplify their long-term well-being.
AB - Survival rates in pediatric hematopoietic stem cell transplantation (HSCT) for nonmalignant diseases have improved due to advances in conditioning regimens, donor selection, and prophylaxis and treatment of infections and graft-versus-host disease. Insight into the long-term patient-reported outcomes (PROs) after pediatric HSCT for nonmalignant disease is lacking but essential for optimal shared decision making, counseling, and quality of care. The purpose of this research was to determine long-term patient-reported outcomes in allogeneic pediatric HSCT for nonmalignant diseases and to compare these results with Dutch reference data. This single-center cohort study evaluated PROs (PedsQL 4.0, PROMIS item banks), self- or proxy-reported, among patients at ?2 years after pediatric allogeneic HSCT for nonmalignant disease. Mean scores were compared with those of the Dutch general population. Of 171 eligible patients, 119 participated, for a 70% response rate. The median patient age was 15.8 years (range, 2 to 49 years), and the median duration of follow-up was 8.7 years (range, 2 to 34 years). Indications for HSCT included inborn errors of immunity (n = 41), hemoglobinopathies (n = 37), and bone marrow failure (n = 41). Compared with reference data, significantly lower scores were found in adolescents (age 13 to 17 years) on the Total, Physical Health, and School Functioning PedsQL subscales. Significantly more Sleep Disturbance was reported in children (age 8 to 18 years). On the other hand, significantly better scores were seen on PROMIS Fatigue (age 5 to 7 years) and Pain Interference (age 8 to 18 years) and, in adults (age 19 to 30 years), on Depressive Symptoms and Sleep Disturbance. This study showed better or comparable very long-term PROs in patients after pediatric HSCT for nonmalignant diseases compared with the reference population. Children and adolescents seem to be the most affected, indicating the need for supportive care to prevent impaired quality of life and, more importantly, to amplify their long-term well-being.
KW - Allogeneic hematopoietic stem cell transplantation
KW - Health-related quality of life
KW - Late effects
KW - Long-term follow-up
KW - Patient-reported outcomes
KW - Pediatric
UR - http://www.scopus.com/inward/record.url?scp=85147119057&partnerID=8YFLogxK
U2 - https://doi.org/10.1016/j.jtct.2022.12.024
DO - https://doi.org/10.1016/j.jtct.2022.12.024
M3 - Article
C2 - 36587742
SN - 2666-6367
VL - 29
SP - 186.e1-186.e10
JO - Transplantation and Cellular Therapy
JF - Transplantation and Cellular Therapy
IS - 3
ER -