Search

Byline: Renu. Suthar, Shivan. Kesavan, Indar. Sharawat, Manisha. Malviya, Titiksha. Sirari, Bhupendra. Sihag, Arushi. Saini, Vishalavath. Jyothi, Naveen. Sankhyan Background: X-linked dystrophinopathies have a wide spectrum of manifestation. The most [...]

Background & objectives: Cause of death assignment from verbal autopsy (VA) questionnaires is conventionally accomplished through physician review. However, since recently, computer softwares have been developed to assign the cause of death. The present study evaluated the performance of computer software in assigning the cause of death from the VA, as compared to physician review. Methods: VA of 600 adult deaths was conducted using open- and close-ended questionnaires in Nandpur Kalour Block of Punjab, India. Entire VA forms were used by two physicians independently to assign the cause of death using the International Statistical Classification of Diseases and Related Health Problems (ICD)-10 codes. In case of disagreement between them, reconciliation was done, and in cases of persistent disagreements finally, adjudication was done by a third physician. InterVA-4-generated causes from close-ended questionnaires were compared using Kappa statistics with causes assigned by physicians using a questionnaire having both open- and close-ended questions. At the population level, Cause-Specific Mortality Fraction (CSMF) accuracy and P-value from McNemar's paired Chi-square were calculated. CSMF accuracy indicates the absolute deviation of a set of proportions of causes of death out of the total number of deaths between the two methods. Results: The overall agreement between InterVA-4 and physician coding was 'fair' (?=0.42; 95% confidence interval 0.38, 0.46). CSMF accuracy was found to be 0.71. The differences in proportions from the two methods were statistically different as per McNemar's paired Chi-square test for ischaemic heart diseases, liver cirrhosis and maternal deaths. Interpretation & conclusions: In comparison to physicians, assignment of causes of death by InterVA-4 was only 'fair'. Hence, it may be appropriate to continue with physician review as the optimal option available in the current scenario. [ABSTRACT FROM AUTHOR]

Background: Duchenne muscular dystrophy (DMD) is a rare progressive muscular disease that primarily affects boys. A lack of comprehensive care for patients living with DMD is directly associated with a compromised quality of life (QoL) for those affected and their caregivers. This disease also has a huge economic impact on families as its treatment requires substantial direct, indirect, and informal care costs., Objective: This study presents a protocol developed to evaluate the feasibility and efficacy of a patient-centered care (PCC) model for children with DMD. The care model was designed with the aim to empower families, improve QoL, and reduce economic burden on their families., Methods: This study is planned as a quasi-experimental study that will enroll 70 consecutive families with boys (aged 5-15 years) with DMD visiting a tertiary care center. The study is being conducted in 2 phases (preintervention and postintervention phases, referred to as phase 1 and phase 2, respectively). During phase 1, the patients received routine care. The study is now in phase 2, with the intervention currently being administered. The intervention is based on the PCC model individualized by the intervention team. The model has a comprehensive DMD telecare component that includes teleconsultation as one of its key components to reduce in-person physician visits at the health facility. Teleconsultation is especially beneficial for late-ambulatory and nonambulatory patients. Data on economic burden are being collected for out-of-pocket expenses for both phases during in-person visits via telephone or messaging apps on a monthly basis. QoL data for patients and their primary caregivers are being collected at 3 time points (ie, time of enrollment, end of phase 1, and end of phase 2). Outcome measures are being assessed as changes in economic burden on families and changes in QoL scores., Results: Participant recruitment began in July 2021. The study is ongoing and expected to be completed by March 2023. The findings based on baseline data are expected to be submitted for publication in 2023., Conclusions: This paper outlines a research proposal developed to study the impact of a PCC model for patients with DMD in low- and middle-income countries (LMICs). This study is expected to provide evidence of whether a multicomponent, patient-centric intervention could reduce economic burdens on families and improve their QoL. The results of this study could guide policy makers and health professionals in India and other LMICs to facilitate a comprehensive care program for patients living with DMD. The economic impact of a rare disease is an important consideration to formulate or evaluate any health policy or intervention related to new treatments and financial support schemes., Trial Registration: Clinical Trials Registry India (ICMR-NIMS) CTRI/2021/06/034274; https://www.ctri.nic.in/Clinicaltrials/pmaindet2.php?trialid=56650., International Registered Report Identifier (irrid): PRR1-10.2196/42491., (©Titiksha Sirari, Renu Suthar, Amarjeet Singh, Shankar Prinja, Vishwas Gupta, Manisha Malviya, Akashdeep Singh Chauhan, Naveen Sankhyan. Originally published in JMIR Research Protocols (https://www.researchprotocols.org), 28.04.2023.)