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

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 care was evaluated by the Measure of Processes of Care questionnaire. Sixty-three HS patients receiving HSCT with at least 3 years of follow-up after HSCT were 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, -0.7 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.

SUBMITTER: Aldenhoven M

PROVIDER: S-EPMC5737136 | biostudies-literature | 2017 Nov

REPOSITORIES: biostudies-literature

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

Aldenhoven Mieke M van den Broek Brigitte T A BTA Wynn Robert F RF O'Meara Anne A Veys Paul P Rovelli Attilio A Jones Simon A SA Parini Rossella R van Hasselt Peter M PM Renard Marleen M Bordon Victoria V de Koning Tom J TJ Boelens Jaap Jan JJ

Blood advances 20171107 24

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 ...[more]

PMID: 29296871

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Project description:During hospitalization for hematopoietic stem cell transplantation (HCT), patients experience a steep deterioration in quality of life (QOL) and mood. To our knowledge, the impact of this deterioration on patients' QOL and posttraumatic stress disorder (PTSD) symptoms after HCT is unknown.We conducted a prospective longitudinal study of patients hospitalized for HCT. They assessed QOL using the Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT) and depression and anxiety symptoms were assessed using the Patient Health Questionnaire-9 (PHQ-9) at the time of admission for HCT, during hospitalization, and 6 months after HCT. We also used the Hospital Anxiety and Depression Scale (HADS) to measure patients' anxiety and depression symptoms at baseline and during HCT hospitalization. The PTSD Checklist was used to assess for PTSD symptoms. Multivariable linear regression models were used to identify predictors of QOL and PTSD symptoms at 6 months.We enrolled 90 of 93 consecutively eligible patients (97%) undergoing autologous and allogeneic HCT. Data at 6 months were available for 67 participants. At 6 months, 28.4% of participants met the criteria for PTSD and 43.3% had clinically significant depression. On multivariable regression analyses adjusting for significant covariates, changes in QOL and depression scores from week 2 of HCT hospitalization to baseline predicted worse QOL (changes in scores between week 2 and baseline [?] QOL: ?, 0.94 [P<.0001] and ? PHQ-9: ?, -2.59 [P = 0.001]) and PTSD symptoms (? QOL: ?, -0.40 [P<.0001] and ? PHQ-9: ?, 1.26 [P<.0001]) at 6 months after HCT.Six months after HCT, a significant percentage of patients met the criteria for PTSD and depression. A decline in QOL and an increase in depressive symptoms during hospitalization for HCT were found to be the most important predictors of 6-month QOL impairment and PTSD symptoms. Therefore, managing symptoms of depression and QOL deterioration during HCT hospitalization may be critical to improving QOL at 6 months and reducing the risk of PTSD. Cancer 2016;122:806-812. © 2015 American Cancer Society.

Project description:BACKGROUND/AIM:Physical function is known to decrease after hematopoietic stem cell transplantation (HSCT), with the most substantial impairment noted at 90 days post-transplantation. Little is known about the natural course of physical function during the acute post-transplant period preciously. The aim of the study was to monitor the changes in physical function through serial evaluations of the physical function, and identify the effect of physical function on QoL during the acute post-transplant period. PATIENTS AND METHODS:This prospective cohort study included 41 patients admitted for planned autologous or allogeneic HSCT. Physical impairment was evaluated with decrease in the de Morton Mobility Index (DEMMI) every week and defined as a DEMMI score of more than 2 points after HSCT. The outcome variables for QoL included visual analogue scale (VAS), European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30), and Zung Self-rating Depression Scale (SDS) at enrollment and discharge. RESULTS:Based on DEMMI scores, 24.40% of all HSCT patients showed physical impairment, for whom the DEMMI score showed an overall decrease during hospitalization with significant differences in scores at 1, 2, and 3 weeks after HSCT, between 1 week before and 3 weeks after HSCT, and between 1 and 3 weeks after HSCT. There was no significant difference of VAS between admission and discharge between the groups. Each functional subscale of EORTC QLQ-C30 differed significantly between the groups, with lower scores in the physical impairment group. There was only a significant difference in SDS at discharge between the groups. QoL pre-transplantation can be a predictive factor for physical impairment during the acute post-transplant period, which can be detected in the early period after HSCT. CONCLUSION:Patients during acute post-transplant period had physical impairment and QoL of pre-transplantation was considered a predictive factor for physical impairment. The physical impairment can be detected in the early period after HSCT. Therefore, monitoring of standardized functional outcome measures is important to prevent physical impairment following HSCT.

Dataset Information

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

Publications

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

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