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Health Related Quality of Life and Emotional Health in Children with Chronic Granulomatous Disease: A Comparison of Those Managed Conservatively with Those That Have Undergone Haematopoietic Stem Cell Transplant

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Abstract

Purpose

Chronic Granulomatous Disease (CGD) is a rare primary immunodeficiency that predisposes to life-threatening infections and inflammation. Haematopoietic stem cell transplant (HSCT) can cure CGD. Chronic illness reduces quality of life. Children with haematological malignancies report improved quality of life post-HSCT. There are no data for children with CGD. This study evaluated quality of life and emotional well-being in CGD children treated conventionally or transplanted.

Methods

Parents and children completed the Pediatric Quality of Life Inventory v4.0 (PedsQL) and Strengths and Difficulties Questionnaires (SDQ). Mean scores were compared with published UK norms. Comparisons were made for those that had or had not undergone HSCT.

Results

Forty-seven parents completed PedsQL (children aged 3–15). Twenty-one were post-HSCT. Forty-two completed SDQ (children aged 3–15). Nineteen post-HSCT. Median age for non-HSCT group 9 years. Median age for post-HSCT group 10 years. The HSCT group were median 3 years post-HSCT (range 1–9 years). HSCT survival was 90 %—two died without completing questionnaires

Parent and self-reported quality of life for non-transplanted children was significantly lower than healthy children. Parents reported increased emotional difficulties compared to published norms. PedsQL and SDQ scores for transplanted children were not significantly different from healthy norms.

Conclusions

This study demonstrates the quality of life is reduced in CGD. Transplanted patients have quality of life comparable to levels reported in healthy children. This data will help inform families and clinicians when deciding about treatment and may have relevance for other immunodeficiencies treated with transplant.

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Abbreviations

CGD:

Chronic granulomatous disease

HSCT:

Haematopoietic stem cell transplant

PedsQL:

Pediatric quality of life inventory

SDQ:

Strengths and difficulties questionnaire

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Acknowledgements

The CGD Society advertised and supported the UK Registry update.

This paper presents independent research funded by the National Institute for Health Research (NIHR) under its Research for Patient Benefit (RfPB) Programme (Grant Reference Number PB-PG-0909-19060). The views expressed are those of the author(s) and not necessarily those of the NHS, the NIHR or the Department of Health.

Conflict of interest

The authors declare no conflict of interest.

Author information

Authors and Affiliations

  1. Institutes of Cellular Medicine, Newcastle University, Newcastle, UK

    Theresa Cole, Andrew J. Cant & Andrew R. Gennery

  2. Health & Society, Newcastle University, Newcastle, UK

    Mark S. Pearce

  3. Department of Health Psychology, Newcastle & North Tyneside NHS Trust, Newcastle, UK

    Fiona McKendrick

  4. Department of Psychosocial Services, Great Ormond Street Hospital, London, UK

    Penny Titman

  5. Clinical Immunology, Great Ormond Street Hospital, London, UK

    Catherine M. Cale

  6. Institute of Child Health, University College London, London, UK

    David Goldblatt

  7. Department of Paediatric Immunology & Infectious Diseases, Old Children’s Outpatients, Royal Victoria Infirmary, Newcastle, NE1 4LP, UK

    Theresa Cole

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  1. Theresa Cole
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  2. Fiona McKendrick
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  3. Penny Titman
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  4. Andrew J. Cant
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  8. Andrew R. Gennery
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Correspondence to Theresa Cole.

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Cole, T., McKendrick, F., Titman, P. et al. Health Related Quality of Life and Emotional Health in Children with Chronic Granulomatous Disease: A Comparison of Those Managed Conservatively with Those That Have Undergone Haematopoietic Stem Cell Transplant. J Clin Immunol 33, 8–13 (2013). https://doi.org/10.1007/s10875-012-9758-0

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  • DOI: https://doi.org/10.1007/s10875-012-9758-0

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