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Sattenapalli et al.: Steroid versus Non-Steroid Regimen in Duchenne Muscular Dystrophy There are no large studies from Southern India describing the treatment regimen of the Duchenne muscular dystrophy which is the most common muscular dystrophy. The current observational study aimed to compare steroid versus non-steroid treatment outcomes in Duchenne muscular dystrophy diagnosed population of South India. Immunohistochemically/genetically confirmed Duchenne muscular dystrophy patients diagnosed between February 2019 and March 2021 were ambispectively included. The main outcomes assessed in the study were loss of ambulation with respect to age, disease progression of motor milestones, medical research council grade, change in body weight and reported adverse events.We correlated these outcomes comparatively for both intervention groups (steroid and non-steroid). A total of 274 Duchenne muscular dystrophy patients were included in the study. The mean minimal age of onset of disease was 2.0±1.2 y. 32.85 % patients were treated with steroids and 67.15 % patients were on non-steroidal treatment. Comparatively, both regimen using patients demonstrated loss of ambulation at 13 y of age. Gait, stairs, Gower, chair scores reported loss of motor functions at 13 y and 25 y for steroid and non-steroid using patients respectively. Medical research council grade improved in steroid using patients to a greater extent compared to non-steroid using patients. Change in body weight and adverse events were comparatively low in non-steroid using patients to that of steroid using patients. The pattern of primary outcome measures in this large cohort is comparable despite variability in medical care. The contemporary observational study adds to the real-world evidence in guiding clinicians towards better approach in treating Duchenne muscular dystrophy patient populationa.

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Background Duchene muscular dystrophy (DMD) is an X-linked progressive muscle disorder that is characterized by proximal muscle weakness followed by a premature death in young boys. There is a low index of reports on diagnosis ratio and clinical features in Southern India. Objective The present study aimed to conduct an observational survey on preliminary analysis, family history, associated complaints, and diagnosis ratio of DMD in southern regions of India. Materials and Methods A systematic observation and survey were conducted on clinically confirmed DMD patients registered between 2019 and 2021 through the questionnaire. The questionnaire and pattern of study were identified by exploring published and unpublished studies available from electronic databases and critical assessment criteria considered by physicians. Preliminary analysis such as onset criteria, motor difficulties, milestone delay; family history and consanguinity analysis; chief complaints (ambulatory status, lordosis, respiratory, and cardiac outcomes), associated complaints such as enlarged tongue, oral hygiene, behavioral problems; and other similar parameters were studied. An assessment of the diagnosis rate and pattern was performed. Statistical analysis The data were reviewed and interpreted through statistical methods mean ± standard deviation represented as a percentage. Results In total, 400 DMD patients were included and 250 participated in the study. The onset age group was 2 to 5 years in 37% of the population. Milestone delay was seen in 86%; consanguinity marriage of parents was reported in 39%. Frequent falls were reported in 62% in 5 to 8 years old group. Wheelchair status was reported in 65% in 9 to 12 years old. Cervical and lumbar lordoses were seen in 57 and 69%, respectively, in above 13 years old. Respiratory and cardiac complications were 88 and 78% reported in above 13 years old, respectively. Other major associated complaints such as enlarged tongue were reported in 79%. Fifty-one percent underwent genetic diagnosis and 79% of the population underwent serum creatine phosphokinase (CPK) analysis for the confirmation of DMD. Conclusion In this study population of South India, milestone delay was a major observation. Although there was a slight margin, family history shows “no blood relation among parents” in the majority of the study population. Chief complaints were predominantly severe above 13-year age group population. Serum CPK was the first choice for the first investigation, which is followed by a genetic diagnosis.

Background: Plumbago zeylanica L. is one of the extremely accessible conventionally used herbal plants with various biological activities. However, actions of P. zeylanica L on blood clotting and other complications of blood were indisposed therapeutically studied. Therefore, the scope of the current exploration is to screen the thrombolytic, antioxidant, and cytotoxic effects of leaf extracts. Materials and Methods: Thrombolytic activity (in vitro) was assessed with clot lysis and thrombin inhibitory ability. Further, thrombolytic activity (in vivo) was evaluated by a thrombotic tail (carrageenan-induced) animal model. DPPH and nitric oxide (free radical) scavenging methods were employed to check the in vitro antioxidant property. Further cytotoxicity and acute oral toxicity were assessed for plant extract. Results: The quantitative analysis elicits the presence of the magnificent amount of the total phenolic content (96.8±7.92 mg GAE/g) and total flavonoid content (63.52±4.54 mg QE/g) on the dry weight basis. The maximum clot lysis (96.83%±0.657) of methanolic leaf extract was detected in in vitro model at 800 µg/mL in 72 hr. A strong thrombin inhibition (94.63±2.12%) effect was observed for methanol leaf extract at 2 mg/mL. In in vivo studies a significant (p< 0.001) clot lysis was achieved at the tested dose (100, 200 and 300 mg/kg). DPPH radical and nitric oxide scavenging activity showed the IC 50 value of 25.47±0.51 and 56.32±0.85, respectively. The methanolic extract was found safer up to the highest lethal dose of 2000 mg/kg. Conclusion: These findings suggested that the plant leaves are comprised of significant thrombolytic properties. It could be a promising source for the existence of antioxidant and thrombolytic agents.