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Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantation

Aldenhoven, Mieke ; Van Den Broek, Brigitte T.A. ; Wynn, Robert F. ; O'Meara, Anne ; Veys, Paul ; Rovelli, Attilio ; Jones, Simon A. ; Parini, Rossella ; Van Hasselt, Peter M. and Renard, Marleen , et al. (2017) In Blood Advances 1(24). p.2236-2242
Abstract

Hurler syndrome (HS) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Hematopoietic stem cell transplantation (HSCT) results in long-term survival, although with significant residual disease burden. How this residual disease affects the health-related quality of life is unknown. Therefore, we conducted a multicenter cohort study on functional and psychosocial health and compared the outcomes to normative data using the Child Health Questionnaire and Pediatric Outcomes Data Collection Instrument. Perception of carewas evaluated by the Measure of Processes of Care questionnaire. Sixty-threeHS patients receivingHSCTwith at least 3 years of follow-up afterHSCTwere included. The influence of... (More)

Hurler syndrome (HS) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Hematopoietic stem cell transplantation (HSCT) results in long-term survival, although with significant residual disease burden. How this residual disease affects the health-related quality of life is unknown. Therefore, we conducted a multicenter cohort study on functional and psychosocial health and compared the outcomes to normative data using the Child Health Questionnaire and Pediatric Outcomes Data Collection Instrument. Perception of carewas evaluated by the Measure of Processes of Care questionnaire. Sixty-threeHS patients receivingHSCTwith at least 3 years of follow-up afterHSCTwere included. The influence of potential predictors was analyzed using linear regression analysis, and correlation analysis was performed using Spearman rank correlation. Functional health of transplanted HS patients was significantly diminished compared with normative data (median physical summary z score, -2.4 [range, -3.5 to -1.6]; median global functioning z score, -3.2 [range, -4.8 to -1.8]). Psychosocial health was comparable or only slightly reduced compared with healthy peers (median psychosocial summary z score, 0.15 [range, 20.7 to 0.8]). A higher obtained lysosomal enzyme level post-HSCT predicted for superior functional health. Overall, parents were satisfied with the care received. Functional health of transplanted HS patients appeared significantly more affected than psychosocial health. To improve functional health, the use of only noncarrier donors and striving to achieve full-donor chimerism, both resulting in higher enzyme levels, is advised. Assessing the health-related quality of life could play an important role in evaluating outcomes of HS patients receiving novel (cell) therapies, including autologous gene-transduced HSCT.

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publishing date
type
Contribution to journal
publication status
published
subject
in
Blood Advances
volume
1
issue
24
pages
7 pages
publisher
American Society of Hematology
external identifiers
  • pmid:29296871
  • scopus:85061080787
ISSN
2473-9529
DOI
10.1182/bloodadvances.2017011387
language
English
LU publication?
no
id
6edfd8ea-20fc-4fd1-b9d1-e2034b3b00a8
date added to LUP
2020-02-26 09:46:28
date last changed
2020-09-23 08:06:41
@article{6edfd8ea-20fc-4fd1-b9d1-e2034b3b00a8,
  abstract     = {<p>Hurler syndrome (HS) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Hematopoietic stem cell transplantation (HSCT) results in long-term survival, although with significant residual disease burden. How this residual disease affects the health-related quality of life is unknown. Therefore, we conducted a multicenter cohort study on functional and psychosocial health and compared the outcomes to normative data using the Child Health Questionnaire and Pediatric Outcomes Data Collection Instrument. Perception of carewas evaluated by the Measure of Processes of Care questionnaire. Sixty-threeHS patients receivingHSCTwith at least 3 years of follow-up afterHSCTwere included. The influence of potential predictors was analyzed using linear regression analysis, and correlation analysis was performed using Spearman rank correlation. Functional health of transplanted HS patients was significantly diminished compared with normative data (median physical summary z score, -2.4 [range, -3.5 to -1.6]; median global functioning z score, -3.2 [range, -4.8 to -1.8]). Psychosocial health was comparable or only slightly reduced compared with healthy peers (median psychosocial summary z score, 0.15 [range, 20.7 to 0.8]). A higher obtained lysosomal enzyme level post-HSCT predicted for superior functional health. Overall, parents were satisfied with the care received. Functional health of transplanted HS patients appeared significantly more affected than psychosocial health. To improve functional health, the use of only noncarrier donors and striving to achieve full-donor chimerism, both resulting in higher enzyme levels, is advised. Assessing the health-related quality of life could play an important role in evaluating outcomes of HS patients receiving novel (cell) therapies, including autologous gene-transduced HSCT.</p>},
  author       = {Aldenhoven, Mieke and Van Den Broek, Brigitte T.A. and Wynn, Robert F. and O'Meara, Anne and Veys, Paul and Rovelli, Attilio and Jones, Simon A. and Parini, Rossella and Van Hasselt, Peter M. and Renard, Marleen and Bordon, Victoria and De Koning, Tom J. and Boelens, Jaap Jan},
  issn         = {2473-9529},
  language     = {eng},
  month        = {11},
  number       = {24},
  pages        = {2236--2242},
  publisher    = {American Society of Hematology},
  series       = {Blood Advances},
  title        = {Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantation},
  url          = {http://dx.doi.org/10.1182/bloodadvances.2017011387},
  doi          = {10.1182/bloodadvances.2017011387},
  volume       = {1},
  year         = {2017},
}