Dataset Information

Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey.

ABSTRACT: Primary Immune Regulatory Disorders (PIRD) are an expanding group of diseases caused by gene defects in several different immune pathways, such as regulatory T cell function. Patients with PIRD develop clinical manifestations associated with diminished and exaggerated immune responses. Management of these patients is complicated; oftentimes immunosuppressive therapies are insufficient, and patients may require hematopoietic cell transplant (HCT) for treatment. Analysis of HCT data in PIRD patients have previously focused on a single gene defect. This study surveyed transplanted patients with a phenotypic clinical picture consistent with PIRD treated in 33 Primary Immune Deficiency Treatment Consortium centers and European centers. Our data showed that PIRD patients often had immunodeficient and autoimmune features affecting multiple organ systems. Transplantation resulted in resolution of disease manifestations in more than half of the patients with an overall 5-years survival of 67%. This study, the first to encompass disorders across the PIRD spectrum, highlights the need for further research in PIRD management.

SUBMITTER: Chan AY

PROVIDER: S-EPMC7046837 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature

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Publications

Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey.

Chan Alice Y AY Leiding Jennifer W JW Liu Xuerong X Logan Brent R BR Burroughs Lauri M LM Allenspach Eric J EJ Skoda-Smith Suzanne S Uzel Gulbu G Notarangelo Luigi D LD Slatter Mary M Gennery Andrew R AR Smith Angela R AR Pai Sung-Yun SY Jordan Michael B MB Marsh Rebecca A RA Cowan Morton J MJ Dvorak Christopher C CC Craddock John A JA Prockop Susan E SE Chandrakasan Shanmuganathan S Kapoor Neena N Buckley Rebecca H RH Parikh Suhag S Chellapandian Deepak D Oshrine Benjamin R BR Bednarski Jeffrey J JJ Cooper Megan A MA Shenoy Shalini S Davila Saldana Blachy J BJ Forbes Lisa R LR Martinez Caridad C Haddad Elie E Shyr David C DC Chen Karin K Sullivan Kathleen E KE Heimall Jennifer J Wright Nicola N Bhatia Monica M Cuvelier Geoffrey D E GDE Goldman Frederick D FD Meyts Isabelle I Miller Holly K HK Seidel Markus G MG Vander Lugt Mark T MT Bacchetta Rosa R Weinacht Katja G KG Andolina Jeffrey R JR Caywood Emi E Chong Hey H de la Morena Maria Teresa MT Aquino Victor M VM Shereck Evan E Walter Jolan E JE Dorsey Morna J MJ Seroogy Christine M CM Griffith Linda M LM Kohn Donald B DB Puck Jennifer M JM Pulsipher Michael A MA Torgerson Troy R TR

Frontiers in immunology 20200221

Primary Immune Regulatory Disorders (PIRD) are an expanding group of diseases caused by gene defects in several different immune pathways, such as regulatory T cell function. Patients with PIRD develop clinical manifestations associated with diminished and exaggerated immune responses. Management of these patients is complicated; oftentimes immunosuppressive therapies are insufficient, and patients may require hematopoietic cell transplant (HCT) for treatment. Analysis of HCT data in PIRD patien ...[more]

PMID: 32153572

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Project description:BackgroundCaregivers of children with primary immunodeficiency disorders (PIDs) experience significant psychological distress during their child's hematopoietic cell transplantation (HCT) process.ObjectivesThis study aims to understand caregiver challenges and identify areas for health care system-level improvements to enhance caregiver well-being.MethodsIn this mixed-methods study caregivers of children with PIDs were contacted in August to November 2017 through online and electronic mailing lists of rare disease consortiums and foundations. Caregivers were invited to participate in an online survey assessing sociodemographic variables, the child's medical characteristics, psychosocial support use, and the World Health Organization-5 Well-Being Index. Open-ended questions about health care system improvements were included. Descriptive statistics and linear multivariate regression analyses were conducted. A modified content analysis method was used to code responses and identify emergent themes.ResultsAmong the 80 caregiver respondents, caregivers had a median age of 34 years (range, 23-62 years) and were predominantly female, white, and married with male children given a diagnosis of severe combined immune deficiency. In the adjusted regression model lower caregiver well-being was significantly associated with lower household income and medical complications. Challenges during HCT include maintaining relationships with partners and the child's healthy sibling or siblings, managing self-care, and coping with feelings of uncertainty. Caregivers suggested several organizational-level solutions to enhance psychosocial support, including respite services, online connections to other PID caregivers, and bedside mental health services.ConclusionsCertain high-risk subpopulations of caregivers might need more targeted psychosocial support to reduce the long-term effect of the HCT experience on their well-being. Caregivers suggested several organizational-level solutions for provision of this support.

Dataset Information

Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey.

Publications

Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey.

Similar Datasets

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