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. 2023 Sep 20:11:1212012.
doi: 10.3389/fped.2023.1212012. eCollection 2023.

Evaluating case management for caregivers of children with spinal muscular atrophy type I and II-an exploratory, controlled, mixed-methods trial

Jana Willems  1 , Astrid Pechmann  2 , Sabine Wider  2 , Rita Ambs  2 , Sylvia A N Meyer  2 , Isabel Cascante  3 , Joachim Sproß  4 , Annette Mund  5 , Erik Farin-Glattacker  1 , Thorsten Langer  1
Affiliations

Evaluating case management for caregivers of children with spinal muscular atrophy type I and II-an exploratory, controlled, mixed-methods trial

Jana Willems et al. Front Pediatr. .

Abstract

Introduction: Spinal muscular atrophy (SMA) is a rare neuromuscular disease requiring various clinical specialists and therapists to provide care. Due to the disease's dynamic nature and the long distances between specialized centers and local providers, integrating care between disciplines can be challenging. Care that is inadequately integrated can compromise the quality of care and become a burden for patients and families. This trial aimed to improve the care of patients through a case management (CM) intervention.

Methods: We conducted an exploratory, controlled, two-arm trial with pre-, post-, and follow-up measures (process and outcome evaluation). Proof of efficacy based on statistical significance was not our primary study objective since we were investigating a rare disease. Primary outcomes were caregivers' HRQoL and caregiver-rated quality of care integration. Our secondary outcome was the children's HRQoL.

Results: Questionnaires and semi-structured interviews yielded heterogeneous results depending on caregivers' level of experience and desire (or possibility) to delegate care tasks.

Discussion: Despite differing perceptions, all participants supported the establishment of a care coordination model. We recommend CM immediately after diagnosis to provide the greatest benefit to families. We hope that our trial will support the further development of CM interventions that can be customized for specific diseases.

Keywords: case management; health-related quality of life; integrated care; rare diseases; spinal muscular atrophy.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

Figure 1
Figure 1
Participant timeline; T0, start of intervention (IG)/recruitment of participants (CG); T1, 6 months after start of intervention/recruitment; T2, 12-months-follow-up-period.
Figure 2
Figure 2
Illustration of mean values in IG and CG for PICS-D scales “team quality and communication” and “family impact” (f.l.t.r.); scale range 1−6; higher scores indicate better perceived care network quality and communication (“Team quality and communication”)/better perceived communication of disease's impact on the family by the HCP (“Family impact”).
Figure 3
Figure 3
Illustration of mean values in IG and CG for faBel total score; scale range 4−16, higher scores indicate higher burden.
Figure 4
Figure 4
Illustration of mean values in IG and CG for SF-12 scales “mental health” and “physical health” (f.l.t.r.); scale range 0−100; higher scores indicate better mental/physical functioning.
Figure 5
Figure 5
Illustration of mean values in IG and CG for pedsQL total score; scale range 0−100; higher scores indicate better HRQoL.
See this image and copyright information in PMC

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