Health-related quality of life in patients with early-detected central congenital hypothyroidism

Standard

In: Journal of clinical endocrinology and metabolism, Vol. 106, No. 10, 01.10.2021, p. E4231-E4241.

Research output: Contribution to journal › Article › Academic › peer-review

BibTeX

@article{364f4c2977374c9ca4d2206e0bc912d8,

title = "Health-related quality of life in patients with early-detected central congenital hypothyroidism",

abstract = "Context: Central congenital hypothyroidism (CH) requires lifelong medical treatment. The majority of children with central CH have multiple pituitary hormone deficiencies (MPHD), but in some cases central CH is isolated. Most pituitary hormone deficiencies are associated with impaired health-related quality of life (HRQoL). However, studies on HRQoL in central CH are lacking. Objective: To evaluate HRQoL and fatigue in children and young adults with central CH, as well as parent perspectives. Design: Nationwide cross-sectional study comparing HRQoL between early-detected central CH patients and unaffected siblings with the Pediatric Quality of Life inventory (PedsQL{\texttrademark}) and PedsQL Multidimensional Fatigue Scale. Participants ≥ 8 years old filled in self-reports; parents of participants aged 3 to 18 years filled in parent reports. Isolated central CH patients, MPHD patients, and siblings were compared using a linear mixed model andTukey{\textquoteright}s post hoc test. Results: Eighty-eight patients and 52 siblings participated, yielding 98 self-reports and 115 parent reports. Isolated central CH patients (n = 35) and siblings showed similar scores on all subscales, both in the self-reports and parent reports. For MPHD patients (n = 53), self-reported scores were similar to those of siblings. Parent reported total HRQoL and fatigue scores were significantly poorer in MPHD patients compared with siblings (mean differences -10.2 and -9.4 points; P < 0.01), as were scores for physical functioning, social functioning and general fatigue. Conclusion: Self-reported HRQoL scores in isolated central CH and MPHD patients were similar to siblings. However, parents reported significantly lower HRQoL and fatigue scores for MPHD patients, suggesting a difference in perceived limitations between MPHD patients and their parents.",

keywords = "Central hypothyroidism, Congenital hypothyroidism, Fatigue, Neonatal screening, Quality of life",

author = "Naafs, {Jolanda C.} and Marchal, {Jan Pieter} and Verkerk, {Paul H.} and Eric Fliers and {van Trotsenburg}, {A. S. Paul} and Nitash Zwaveling-Soonawala",

note = "Funding Information: NZS has received a grant from Pfizer to support this investigator-initiated study (tracking number WI219179). Pfizer was not involved in patient recruitment, data collection, data analysis, or preparation of the manuscript. Publisher Copyright: {\textcopyright} The Author(s) 2021.",

year = "2021",

month = oct,

day = "1",

doi = "10.1210/clinem/dgab209",

language = "English",

volume = "106",

pages = "E4231--E4241",

journal = "Journal of clinical endocrinology and metabolism",

issn = "0021-972X",

publisher = "The Endocrine Society",

number = "10",

}

RIS

TY - JOUR

T1 - Health-related quality of life in patients with early-detected central congenital hypothyroidism

AU - Naafs, Jolanda C.

AU - Marchal, Jan Pieter

AU - Verkerk, Paul H.

AU - Fliers, Eric

AU - van Trotsenburg, A. S. Paul

AU - Zwaveling-Soonawala, Nitash

N1 - Funding Information: NZS has received a grant from Pfizer to support this investigator-initiated study (tracking number WI219179). Pfizer was not involved in patient recruitment, data collection, data analysis, or preparation of the manuscript. Publisher Copyright: © The Author(s) 2021.

PY - 2021/10/1

Y1 - 2021/10/1

N2 - Context: Central congenital hypothyroidism (CH) requires lifelong medical treatment. The majority of children with central CH have multiple pituitary hormone deficiencies (MPHD), but in some cases central CH is isolated. Most pituitary hormone deficiencies are associated with impaired health-related quality of life (HRQoL). However, studies on HRQoL in central CH are lacking. Objective: To evaluate HRQoL and fatigue in children and young adults with central CH, as well as parent perspectives. Design: Nationwide cross-sectional study comparing HRQoL between early-detected central CH patients and unaffected siblings with the Pediatric Quality of Life inventory (PedsQL™) and PedsQL Multidimensional Fatigue Scale. Participants ≥ 8 years old filled in self-reports; parents of participants aged 3 to 18 years filled in parent reports. Isolated central CH patients, MPHD patients, and siblings were compared using a linear mixed model andTukey’s post hoc test. Results: Eighty-eight patients and 52 siblings participated, yielding 98 self-reports and 115 parent reports. Isolated central CH patients (n = 35) and siblings showed similar scores on all subscales, both in the self-reports and parent reports. For MPHD patients (n = 53), self-reported scores were similar to those of siblings. Parent reported total HRQoL and fatigue scores were significantly poorer in MPHD patients compared with siblings (mean differences -10.2 and -9.4 points; P < 0.01), as were scores for physical functioning, social functioning and general fatigue. Conclusion: Self-reported HRQoL scores in isolated central CH and MPHD patients were similar to siblings. However, parents reported significantly lower HRQoL and fatigue scores for MPHD patients, suggesting a difference in perceived limitations between MPHD patients and their parents.

AB - Context: Central congenital hypothyroidism (CH) requires lifelong medical treatment. The majority of children with central CH have multiple pituitary hormone deficiencies (MPHD), but in some cases central CH is isolated. Most pituitary hormone deficiencies are associated with impaired health-related quality of life (HRQoL). However, studies on HRQoL in central CH are lacking. Objective: To evaluate HRQoL and fatigue in children and young adults with central CH, as well as parent perspectives. Design: Nationwide cross-sectional study comparing HRQoL between early-detected central CH patients and unaffected siblings with the Pediatric Quality of Life inventory (PedsQL™) and PedsQL Multidimensional Fatigue Scale. Participants ≥ 8 years old filled in self-reports; parents of participants aged 3 to 18 years filled in parent reports. Isolated central CH patients, MPHD patients, and siblings were compared using a linear mixed model andTukey’s post hoc test. Results: Eighty-eight patients and 52 siblings participated, yielding 98 self-reports and 115 parent reports. Isolated central CH patients (n = 35) and siblings showed similar scores on all subscales, both in the self-reports and parent reports. For MPHD patients (n = 53), self-reported scores were similar to those of siblings. Parent reported total HRQoL and fatigue scores were significantly poorer in MPHD patients compared with siblings (mean differences -10.2 and -9.4 points; P < 0.01), as were scores for physical functioning, social functioning and general fatigue. Conclusion: Self-reported HRQoL scores in isolated central CH and MPHD patients were similar to siblings. However, parents reported significantly lower HRQoL and fatigue scores for MPHD patients, suggesting a difference in perceived limitations between MPHD patients and their parents.

KW - Central hypothyroidism

KW - Congenital hypothyroidism

KW - Fatigue

KW - Neonatal screening

KW - Quality of life

UR - http://www.scopus.com/inward/record.url?scp=85115612776&partnerID=8YFLogxK

U2 - 10.1210/clinem/dgab209

DO - 10.1210/clinem/dgab209

M3 - Article

C2 - 33780546

VL - 106

SP - E4231-E4241

JO - Journal of clinical endocrinology and metabolism

JF - Journal of clinical endocrinology and metabolism

SN - 0021-972X

IS - 10

ER -

ID: 20234201

Generic Portal