JMIR Publications

ABSTRACT

Background:

Duchenne muscular dystrophy is progressive, care is often compromised due to the lack of effective treatment and supportive care. Evolution in the knowledge about disease has changed the natural history of the disease. Early diagnosis and interventions have prolonged survival. A lack of comprehensive care for patients living with disease is directly associated with a compromised quality of life for patients and their caregivers. The disease also has a huge economic impact on families as its treatment requires substantial direct, indirect and informal care costs. There is an urgent need to develop an effective patient-centered care model (PCC), in low- and middle-income countries (LMIC) to improve the QoL of affected families.

Objective:

The study presents a protocol developed to evaluate the PCC model for children with DMD and to improve quality of life and reduce economic burden on the affected patients and their families.

Methods:

The present study is a Quasi-experimental study, in which 70 families with 5–15-year-old boys with DMD registered at Pediatric Neurology Clinic, in a tertiary care center and under regular follow-up would be enrolled consecutively as per footfall. Intervention would be based on PCC model individualized by the intervention team. The total duration of the study would be 18 months, comprising two phases of 6 months each. Six months would be expected period for patient enrolment. First phase, patients will be observed for routine hospital care. Second phase, intervention will be given. First primary outcome would be change in mean scores of QoL of both patients and caregivers in phase 1 and phase 2. The second, outcome would be to assess change in economic impact in terms of out-of-pocket (OOP) expenditure. Apart from the outcomes listed above a PCC model i.e. Comprehensive DMD -Telecare Model would also be developed. Model would include teleconsultation as one of its key components to reduce patient physical visits Teleconsultation would be especially beneficial for non-ambulatory patients.

Results:

Study is ongoing and expected to be completed by March 2023.

Conclusions:

In addition, to sever physical impairment, the disease also has a socio-economic and psychological impact. This study is expected to provide evidence whether a multi-component, patient-centric intervention could reduce the economic burden on families and improve their QoL. Public health significance DMD still remains an orphan disease. The study might guide policymakers and health professionals, in India, and other LMIC to facilitate a comprehensive program for overall care of patients living with DMD. The disability is not merely an individual impairment but more so a social phenomenon. This “care” approach will generate awareness in society. Later, acceptance of the illness and the affected families. The economic impact of a rare disease is a important consideration to formulate or evaluate any health policy or intervention related to new treatments and financial support schemes. Clinical Trial: CTRI/2021/06/034274.