Diagnostic accuracy of Magnetic Resonance Parkinsonism Index in differentiating progressive supranuclear palsy from Parkinson's disease and controls in Indian patients.

AIMS AND OBJECTIVES: An assessment of the sensitivity and specificity of magnetic resonance (MR) imaging measurements of midbrain, pons, middle cerebellar peduncles (MCPs), and superior cerebellar peduncles (SCPs) and MR Parkinsonism Index (MRPI) in differentiating progressive supranuclear palsy (PSP) from Parkinson's disease (PD) and controls was performed. The correlation of these MR imaging measurements with the duration and severity of disease in the Indian patients using the PSP rating scale (PSPRS) was also performed. MATERIALS AND METHODS: Twenty-six consecutive patients were enrolled in this study, satisfying the diagnostic criteria by the National Institute for Neurological Disorders and Stroke, and the Society for PSP (NINDS-SPSP), along with 13 PD and 30 control patients. All PSP patients were assessed using the PSP rating scale and staging system. Radiologists were blinded to the clinical diagnoses. MRPI was calculated by multiplying the pons area/midbrain area ratio by MCP width/SCP width ratio. The midbrain/pons area (M/P) ratio was measured as the ratio of midbrain area to pons area. RESULTS: Mean MRPI in PSP patients (23.48 ± 9.61) was significantly higher than that in PD patients (9.07 ± 2.23) and controls (9.45 ± 1.87). In this study, MRPI was 100% sensitive, specific, and accurate in differentiating PSP from PD and was 96.3% sensitive, 100% specific, and 98.21% accurate in differentiating PSP from controls. No correlation was found between the duration of disease, PSP rating scale, PSP staging system, and MRPI in the present study. MRPI was only marginally superior to the M/P ratio in differentiating between PSP and PD patients on an individual basis. No overlapping values were observed in the PSP and PD patients. CONCLUSION: Magnetic Resonance Parkinsonism Index is more sensitive, specific, and accurate in differentiating PSP from PD in the early stages on an individual basis.

Low-dose emicizumab prophylaxis in patients with severe hemophilia A: a retrospective study bringing new hope for our patients.

BACKGROUND: Low-dose emicizumab can potentially offer a cost-effective treatment option in persons with hemophilia A, especially in developing countries. OBJECTIVES: To compare the efficacy and safety of low-dose emicizumab with those on low-dose factor (F)VIII prophylaxis via chart review. METHODS: After ethics approval, chart data of 2 groups of patients were reviewed: group 1 (low-dose emicizumab, n = 10; 3 mg/kg monthly without a loading dose) and group 2 (low-dose FVIII prophylaxis, n = 10; 10-20 IU/kg of FVIII concentrates twice a week). Outcomes were target joints, annual bleeding rate, annual joint bleeding rate, Hemophilia Joint Health Score, nonactivated thromboelastometry-rotational thromboelastometry clotting time, plasma emicizumab levels, and direct costs of treatment. RESULTS: All outcome measures were significantly better in the low-dose emicizumab group than in the low-dose FVIII prophylaxis group. For nonactivated thromboelastometry-rotational thromboelastometry, median values after 6 months in the low-dose emicizumab group were comparable with values seen in patients with mild hemophilia, while the values in the low-dose FVIII prophylaxis group were similar to those of patients with moderate hemophilia. The direct cost of low-dose emicizumab was found to be approximately US $6000 and that for low-dose recombinant FVIII prophylaxis used in our study was US $6282 (the cost may range from US $3432 to $7920 depending on the type of factor) when compared to approximately US $15 000 for standard-dose emicizumab. CONCLUSION: Low-dose emicizumab offers a cost-effective treatment option and can improve access in developing countries. These findings need to be confirmed in a larger and better-controlled study.