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. 2019 Mar 2:7:2050312119834470.
doi: 10.1177/2050312119834470. eCollection 2019.

Needs management in families affected by childhood-onset dystrophinopathies

Kristin M Conway  1 , Katy Eichinger  2 , Christina Trout  3 , Paul A Romitti  1 , Katherine D Mathews  4 , Shree K Pandya  2 ; Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet)
Affiliations

Needs management in families affected by childhood-onset dystrophinopathies

Kristin M Conway et al. SAGE Open Med. .

Abstract

Purpose: To collect information about the needs of families affected by childhood-onset dystrophinopathies residing in the United States.

Methods: Individuals with an eligible dystrophinopathy were identified by the Muscular Dystrophy Surveillance, Tracking, and Research network. Between September 2008 and December 2012, 272 caregivers completed a 48-item survey about needs related to information, healthcare services, psychosocial issues, finances, caregiver demographics, and the individual's functioning.

Results: Overall, at least 80% of the survey items were identified as needs for more than one-half of caregivers. Among the needs identified, physical health and access to information were currently managed for most caregivers. Items identified as needed but managed less consistently were funding for needs not covered by insurance and psychosocial support.

Conclusions: Healthcare providers, public health practitioners, and policymakers should be aware of the many needs reported by caregivers, and focus on addressing gaps in provision of needed financial and psychosocial services.

Keywords: Becker muscular dystrophy; Duchenne muscular dystrophy; caregivers; dystrophinopathy; needs assessment; survey.

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Conflict of interest statement

Declaration of conflicting interests: K.D.M. receives funding from National Institutes of Health (NIH) grant 2 U54 NS053672-11, the Friedreich’s Ataxia Research Alliance, the Centers for Disease Control and Prevention (DD000189), PTC Therapeutics Inc, Sarepta Therapeutics Inc, Pfizer Inc, FibroGen Inc, AMO, BMS, Santhera, Acceleron, Takeda, Reata, and Intalfarmaco. Previously, funding was received from GlaxoSmithKline, Eli Lilly and Company, Prosensa Therapeutics BV/BioMarin Pharmaceutical Inc, ViroPharma Inc, Marathon Pharmaceuticals LLC, aTyr Pharma Inc, and Horizon Pharma Ireland Ltd. K.D.M. is an advisory board member for Sarepta Therapeutics Inc and Santhera. K.D.M. is a scientific board member for the Muscular Dystrophy Association and the FSH Society. K.D.M. received no personal funding aside from travel expense reimbursement. The remaining authors (K.M.C., K.E., C.T., P.A.R., and S.K.P.) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.

Figures

Figure 1.
Figure 1.
Flowchart of eligible caregivers and completed questionnaires. *Ineligible caregivers included those of deceased males or those of males with possible or asymptomatic diagnoses, or affected females. **Non-contacts include mailings to an unverified address that were not verified, returned mail with undeliverable address, and unverifiable address for mailings.
See this image and copyright information in PMC

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