Bone density and bone health alteration in boys with Duchenne Muscular Dystrophy: a prospective observational study

Suthar, Renu; Reddy, B. V. Chaithanya; Malviya, Manisha; Sirari, Titiksha; Attri, Savita Verma; Patial, Ajay; Tageja, Minni; Didwal, Gunjan; Khandelwal, Niranjan; Saini, Arushi Gahlot; Saini, Lokesh; Sahu, Jitendra Kumar; Dayal, Devi; Sankhyan, Naveen

doi:10.1515/jpem-2020-0680

Cited by 10 publications

(9 citation statements)

References 39 publications

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“…Suthar et al. ( 28 ) reported a reduction in BMD measured with DXA (height adjusted Z score ≤ −2) in 57% of boys with DMD in North India, which was greater than that observed in our study. Aparicio et al.…”

Section: Discussioncontrasting

confidence: 85%

Bone Mineral Density Assessment by Quantitative Computed Tomography in Glucocorticoid-Treated Boys With Duchenne Muscular Dystrophy: A Linear Mixed-Effects Modeling Approach

et al. 2022

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ObjectiveBoys with Duchenne muscular dystrophy (DMD) are at risk of bone damage and low bone mineral density (BMD). The aim of the study is to examine lumbar BMD values measured by QCT and identify the factors associated with BMD loss using a multilevel mixed-effects model.MethodsLumbar BMD was evaluated by quantitative computed tomography (QCT) at diagnosis, 1 and 2 years follow up in patients with DMD who were treated with GC. Demographic data, functional activity scores (FMSs), laboratory parameters and steroid use were recorded. A multilevel mixed-effects model was used to analyze BMD loss.ResultsNineteen patients with DMD who had a total of sixty complete records between January 2018 and October 2021 were retrospectively analyzed. At baseline, 15.8% of patients (3/19) had low lumbar BMD (Z score ≤ −2), and the mean BMD Z score on QCT was -0.85 (SD 1.32). The mean BMD Z score at 1 and 2 years postbaseline decreased to -1.56 (SD 1.62) and -2.02 (SD 1.36), respectively. In our model, BMD Z score loss was associated with age (β=-0.358, p=0.0003) and FMS (β=-0.454, p=0.031). Cumulative GC exposure and serum levels of calcium, phosphorus, 25(OH)-vitamin D and creatinine kinase did not independently predict BMD loss.ConclusionsThis study demonstrates that in DMD patients, lumbar BMD decreased gradually and progressively. Age and FMS are the main contributors to BMD loss in boys with DMD. Early recognition of risk factors associated with BMD loss may facilitate the development of strategies to optimize bone health.

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Section: Discussioncontrasting

confidence: 85%

Bone Mineral Density Assessment by Quantitative Computed Tomography in Glucocorticoid-Treated Boys With Duchenne Muscular Dystrophy: A Linear Mixed-Effects Modeling Approach

et al. 2022

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“…This includes diagnostic services, physical therapy, orthotics, respiratory therapy, corrective orthopedic surgery, ventilation, feeding support, and speech therapy. Muscle degeneration can be slowed down with the use of corticosteroids, while antibiotics may be needed for respiratory infections [ 8 - 11 ]. Occupational therapy, assistive devices, and a wheelchair are also beneficial to the children.…”

Section: Introductionmentioning

confidence: 99%

Development and Economic Evaluation of a Patient-Centered Care Model for Children With Duchenne Muscular Dystrophy: Protocol for a Quasi-Experimental Study

Sirari¹,

Suthar²,

Singh³

et al. 2023

JMIR Res Protoc

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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

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“…However, glucocorticoid therapy predisposes osteoporosis by decreasing bone formation and increasing bone resorption, growth, and pubertal delay. The proximal femur and spinal vertebrae in boys with DMD show markedly reduced bone mineral density (BMD) [6].…”

Section: Introductionmentioning

confidence: 99%

A portrait of the bone mineral density of boys with Duchenne muscular dystrophy

Vale

Mizumoto

Lima

et al. 2022

Preprint

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Introduction Duchenne muscular dystrophy (DMD) patients are at risk of developing conditions that can compromise their bone health, such as fractures and walking impairment. We aimed to assess bone mineral density as a function of the age of DMD patients. Materials and Methods The cross-sectional study included 48 participants. Bodyweight and height were collected to determine the height-for-age and BMI-for-age Z-scores. Lumbar spine (LS) and total body (TBLH) bone mineral density (BMD) measurements were performed with dual-energy X-ray absorptiometry. Height-for-age Z-scores (HAZ) of boys under 19 years-old were determined, and the BMD was adjusted for HAZ. Results The DMD patients' HAZ curve is on the left compared to the WHO standard. The older patients had lower LS-BMD (p = 0.012) and TBLH-BMD (p <0.001) Z-score values than younger patients. There was a moderate negative correlation between age and the LS-BMD Z-score (ρ = -0.49, p < 0.001), a strong negative correlation between age and the TBLH-BMD Z-score (ρ = -0.82, p < 0.001), and a moderate positive correlation between age and the TBLH-BMDHAZ (ρ = 0.43, p = 0.007). Conclusion For clinical evaluation DMD population, it is necessary to consider the variables related to height since the delay in linear growth may underestimate the BMD Z-scores. DMD patients have impaired linear growth, and older boys could have more severe bone morbidity.

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Bone density and bone health alteration in boys with Duchenne Muscular Dystrophy: a prospective observational study

Cited by 10 publications

References 39 publications

Bone Mineral Density Assessment by Quantitative Computed Tomography in Glucocorticoid-Treated Boys With Duchenne Muscular Dystrophy: A Linear Mixed-Effects Modeling Approach

Bone Mineral Density Assessment by Quantitative Computed Tomography in Glucocorticoid-Treated Boys With Duchenne Muscular Dystrophy: A Linear Mixed-Effects Modeling Approach

Development and Economic Evaluation of a Patient-Centered Care Model for Children With Duchenne Muscular Dystrophy: Protocol for a Quasi-Experimental Study

A portrait of the bone mineral density of boys with Duchenne muscular dystrophy

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