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J Clin Immunol. 2019 Nov;39(8):786-794. doi: 10.1007/s10875-019-00689-2. Epub 2019 Oct 16.

Quality of Life of Patients with Wiskott Aldrich Syndrome and X-Linked Thrombocytopenia: a Study of the Primary Immune Deficiency Consortium (PIDTC), Immune Deficiency Foundation, and the Wiskott-Aldrich Foundation.

Author information

1
Division of Stem Cell Transplantation and Regenerative Medicine, Lucille Packard Children Hospital, Stanford School of Medicine, Stanford, USA. Ashah5@stanford.edu.
2
Lifespan Cancer Institute, Alpert Medical School of Brown University, Providence, USA.
3
Division of Biostatistics, Medical College of Wisconsin, Milwaukee, USA.
4
Wiskott-Aldrich Foundation, Smyrna, USA.
5
Immune Deficiency Foundation, Towson, USA.
6
Department of Psychiatry, University of California-San Francisco, San Francisco, USA.
7
Hauner University Children's Hospital, Ludwig-Maximilians Universitat Munich Germany, Munich, Germany.
8
Division of Allergy, Immunology and Blood and Marrow Transplantation, Benioff Children's Hospital, University of California-San Francisco, San Francisco, USA.

Abstract

BACKGROUND:

We undertook a study to determine the impact of Wiskott Aldrich Syndrome (WAS) and X-linked thrombocytopenia (XLT) and their therapies upon the health-related quality of life (HRQOL) of patients and their families.

MATERIALS AND METHODS:

We undertook a survey of patients and their families, who self-identified as having either WAS or XLT. We assessed the PedsQL™ 4.0, the parent proxy form, and the family impact module. These results were compared with normative data from previously published reports.

RESULTS:

Sixty-eight patients (29 patients completed both the PedsQL™ 4.0 and the parent proxy form; 21 completed only the PedsQL™ 4.0; and 18 completed only the parent proxy form) were included. In contrast to patient-reported outcomes, parents of patients who had a bone marrow transplant (BMT) reported that their children had better QOL scores compared with those who did not (82.6 vs. 73.3, p = 0.023). The QOL of patients vs. previously published normative data showed decreases in patient scores for psychosocial health (72.62 vs. 86.58, p = < 0.001), emotional functioning (69.91 vs. 82.64, p = < 0.001), social functioning (77.55 vs. 91.56, p = < 0.001), and school functioning (70.46 vs. 85.67, p = < 0.001). The family impact study revealed deficits in emotional, social, and cognitive functioning, communication, and worry.

CONCLUSION:

These results show that patients with WAS/XLT are significantly impacted with respect to QOL. BMT offered a better QOL for patients according to parents, but not as reported by the patients. Future studies should incorporate QOL to provide more data and a better understanding of outcomes for long-term survivors and decision-making regarding BMT.

KEYWORDS:

Wiskott Aldrich Syndrome (WAS); X-linked thrombocytopenia (XLT); bone marrow transplant (BMT); quality of life (QOL)

PMID:
31620947
DOI:
10.1007/s10875-019-00689-2

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