Coping with small populations of patients in clinical trials

O’Connor, Daniel; Hemmings, Robert

doi:10.1517/21678707.2014.931221

Cited by 10 publications

(5 citation statements)

References 6 publications

(5 reference statements)

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“…Although recently there have been significant gains in biomedical research being conducted into rare diseases [ 6 ], very few social studies exist which focus on the supportive care needs of parents caring for a child with a rare disease. Studies that have, are typically restricted to a single disease/group of related diseases, involve small sample sizes and are limited to a specific country or culture [ 15 ]. Of the studies which have attempted to identify the supportive care needs of parents in a comprehensive fashion, these have related to paediatric cancers [ 16 , 17 ].…”

Section: Introductionmentioning

confidence: 99%

The supportive care needs of parents with a child with a rare disease: results of an online survey

et al. 2016

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BackgroundParents caring for a child affected by a rare disease have unmet needs, the origins of which are complex and varied. Our aim was to determine the supportive care needs of parents caring for a child with a rare disease.MethodsAn online survey was developed consisting of 45 questions (108 items) and separated into six domains. The survey included questions about perceived level of satisfaction with receiving care, experiences and needs of providing daily care, the impacts of disease on relationships, the emotional and psychological burdens of disease, and parents overall satisfaction with the support received.ResultsThree-hundred and one parents from Australia and New Zealand completed the survey; 91 % (n = 275/301) were mothers, with 132 distinct rare diseases being reported. Fifty-four percent (n = 140/259) of parents were dissatisfied with health professionals’ level of knowledge and awareness of disease; 71 % (n = 130/183) of parents felt they received less support compared to other parents. Information regarding present (60 %, n = 146/240) and future services (72 %, n = 174/240) available for their child were considered important. Almost half of parents (45 %, n = 106/236) struggled financially, 38 % (n = 99/236) reduced their working hours and 34 % (n = 79/236) ceased paid employment. Forty-two percent (n = 99/223) of parents had no access to a disease specific support group, and 58 % (n = 134/230) stated that their number of friends had reduced since the birth of their child; 75 % (n = 173/230) had no contact with other parents with a child with a similar disease, and 46 % (n = 106/230) reported feeling socially isolated and desperately lonely. Most frequent emotions expressed by parents in the week prior to completing the survey were anxiety and fear (53 %, n = 119/223), anger and frustration (46 %, n = 103/223) and uncertainty (39 %, n = 88/223).ConclusionThis study is the first to develop an online survey specifically for use with parents to investigate their supportive care needs across a large and diverse group of rare diseases. The findings highlight that parents with a child with a rare disease have common unmet needs regardless of what disease their child has. Such information may allow health providers to improve child outcomes through improving parental supportive care.

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Section: Introductionmentioning

confidence: 99%

The supportive care needs of parents with a child with a rare disease: results of an online survey

et al. 2016

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“…Out of 20 mothers, seven consented to participate. Given the difficulty in recruiting a sufficient number from populations associated with rare diseases (O'Connor & Hemmings, 2014), we also used a snowball sampling technique, and nine mothers agreed to participate. In total, 16 mothers from eight different prefectures consented to participate, and each of them was interviewed once.…”

Section: Methodsmentioning

confidence: 99%

Mothers' experiences of parenting a child with chromosomal structural abnormalities: The journey to acceptance

Kutsunugi

Tsujino

Murakami

et al. 2020

Japan Journal Nursing Sci

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Purpose This study aimed to investigate the process mothers go through in coming to terms with raising a child with chromosomal structural abnormalities. Methods Sixteen mothers living in Japan were interviewed and a modified grounded theory approach was used for the analysis. Results A total of 35 concepts, nine subcategories, and six categories were extracted. The six categories were: (a) Concern about abnormalities; (b) A healthy child is considered as a standard; (c) Deepening attachment to the child; (d) Acceptance of the child as s/he is; (e) Changing attitude toward disabilities; (f) Creating a frontier for other mothers. The parenting journey meant that parents did not move in a straightforward way from the beginning of the process to the endpoint but instead moved between “Deepening attachment to the child” and “Acceptance of the child as s/he is” before they moved ahead. Conclusion Having support and meeting peers of mothers with similar issues is essential for mothers to review their perspectives that healthy children are the standard against which to measure their child and to motivate them to raise their children, but it was extremely difficult to have such opportunities due to rarity of the disorder. It is crucial to accumulate more practical information so that mothers can access and use it. Mothers also need support to enhance their self‐worth while giving due consideration to the possibility that they may be conscious of being stigmatized. Nurses need to advocate for these children and families to get the appropriate help, understanding and support.

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“…Novel design and analysis approaches for small populations are expected to be beneficial in clinical research aimed at improving care for patients [9, 10]. A deeper understanding of the properties of such designs needs research as well as lessons learned from actual application.…”

Section: Deeper Understanding Of Novel Trial Designsmentioning

confidence: 99%

A framework: make it useful to guide and improve practice of clinical trial design in smaller populations

Roes

2016

BMC Med

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The increased attention to design and analysis of randomised clinical trials in small populations has triggered thinking regarding the most appropriate design methods for a particular clinical research question. Decision schemes and algorithms have been proposed, with varying starting points and foci. Parmar et al. (BMC Medicine 14:183, 2016) proposed a framework designed to assist the clinical trial team in design choices during protocol preparation. Herein, further stimulus is given regarding the extent to which a framework may help change practice for the better, the careful considerations for changing the usual error levels applied and the room for innovation in clinical trial design.Please see related article: http://bmcmedicine.biomedcentral.com/articles/10.1186/s12916-016-0722-3.

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Coping with small populations of patients in clinical trials

Cited by 10 publications

References 6 publications

The supportive care needs of parents with a child with a rare disease: results of an online survey

The supportive care needs of parents with a child with a rare disease: results of an online survey

Mothers' experiences of parenting a child with chromosomal structural abnormalities: The journey to acceptance

A framework: make it useful to guide and improve practice of clinical trial design in smaller populations

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