A randomized study of very low-dose factor VIII prophylaxis in severe haemophilia - A success story from a resource limited country.

INTRODUCTION: Current factor prophylaxis strategy practised in developed countries is not feasible in resource constraint developing countries like India. AIM: The aim of this study was to investigate the efficacy and safety of very low-dose factor prophylaxis in India. METHODS: Children of 1-10 years of age with severe haemophilia were randomized to Prophylaxis group and Episodic (On demand) group. Children in prophylaxis group received very low-dose factor VIII (FVIII) concentrate, i.e. 10 units kg(-1) body weights on 2 days a week. Episodic group received factor concentrate in standard recommended doses. The study period was 11.5 months. RESULTS: In total 21 children were enrolled in this study, 11 assigned to prophylaxis and 10 to episodic group. Children on prophylaxis had 11 joint bleeds in comparison to 57 joint bleeds in episodic group. Mean number of haemarthrosis per patient per month were 0.08 (0.08 ± 0.13) in prophylaxis group compared to 0.48 (0.48 ± 0.34) in episodic group (P < 0.05). Total FVIII consumption was 87.51 and 56.32 units kg(-1) month(-1) in prophylaxis and episodic group respectively (P = ns). Overall median hospital emergency visits were 1 day in prophylaxis group and 9 days in episodic group (P ≤ 0.05). Median days of absenteeism from school were 25 days in episodic group and 3 days in prophylaxis group (P < 0.05). No significant complications were noted in prophylaxis group and compliance was 98%. CONCLUSION: To conclude, low-dose FVIII prophylaxis is cost effective, efficacious and a safe method of preventing joint bleeds and consequent joint damages.

Validation of Risk Stratification for Cardiac Events in Pregnant Women With Valvular Heart Disease.

BACKGROUND: Most risk stratification tools for pregnant patients with heart disease were developed in high-income countries and in populations with predominantly congenital heart disease, and therefore, may not be generalizable to those with valvular heart disease (VHD). OBJECTIVES: The purpose of this study was to validate and establish the clinical utility of 2 risk stratification tools-DEVI (VHD-specific tool) and CARPREG-II-for predicting adverse cardiac events in pregnant patients with VHD. METHODS: We conducted a cohort study involving consecutive pregnancies complicated with VHD admitted to a tertiary center in a middle-income setting from January 2019 to April 2022. Individual risk for adverse composite cardiac events was calculated using DEVI and CARPREG-II models. Performance was assessed through discrimination and calibration characteristics. Clinical utility was evaluated with Decision Curve Analysis. RESULTS: Of 577 eligible pregnancies, 69 (12.1%) experienced a component of the composite outcome. A majority (94.7%) had rheumatic etiology, with mitral regurgitation as the predominant lesion (48.2%). The area under the receiver-operating characteristic curve was 0.884 (95% CI: 0.844-0.923) for the DEVI and 0.808 (95% CI: 0.753-0.863) for the CARPREG-II models. Calibration plots suggested that DEVI score overestimates risk at higher probabilities, whereas CARPREG-II score overestimates risk at both extremes and underestimates risk at middle probabilities. Decision curve analysis demonstrated that both models were useful across predicted probability thresholds between 10% and 50%. CONCLUSIONS: In pregnant patients with VHD, DEVI and CARPREG-II scores showed good discriminative ability and clinical utility across a range of probabilities. The DEVI score showed better agreement between predicted probabilities and observed events.

Functional Status in Hemodialysis - A Comparative Study with FIM, ADLQ and 7D5L Instruments.

There is only limited information on the functional status (FS) of patients receiving hemodialysis (HD) from developing countries where the etiology of chronic kidney disease (CKD) and demographic profile are different. The study aims to assess the FS in patients with CKD using three validated generic instruments. A total of 116 adult patients on HD with a dialysis vintage >3 months were enrolled. FS was assessed using three generic tools - Functional Independence Measure (FIM) (scores 18-126), Seven domains and five levels (7D5L) (scores 0-28), and Activities of Daily Living Questionnaire (ADLQ) (scores 0-6) scales. A higher FIM and ADLQ scores and lower 7D5L score indicated good FS. The mean age of patients was 41.28 ± 15.44 years. About 77.6% were males and 80.2% were unemployed. About 67.2% were receiving twice-weekly HD, and 28.4% were receiving thrice-weekly dialysis. The mean scores were 119.05 ± 11.42 with FIM, 6.44 ± 4.26 with 7D5L, and 5.51 ± 1.19 with ADL instruments. More than 80% showed full functional independence with ADLQ instrument. With FIM, the overall scores showed a tendency for functional independence, but the subdomains involving locomotion/mobility were impaired to a greater extent. The proportion of patients with full independence was less with 7D5L. We observed an inferior FS in individuals <40 years. Patients on HD were functionally independent as assessed by FIM and ADLQ instruments. However, 7D5L appeared to be better in identifying mild to moderate limitations in daily activities. The domains involving motor tasks seem to be affected to a greater extent. The current scales for assessing FS do not incorporate a time-dependent component.

Assessment of health-related quality of life and its determinants in patients with chronic kidney disease.

Health-related quality of life is an important, yet neglected aspect of chronic kidney disease (CKD) care. We evaluated the quality of life and its determinants across CKD 3 to 5D using a kidney disease specific tool (Kidney Disease Quality of Life-SF™) in an underprivileged, predominantly rural population with high rates of illiteracy and unemployment. The scores of individual domains were summarized to three composite scores - physical composite summary (PCS), mental composite summary (MCS), and kidney disease component summary score (KDCS). A total number of 204 participants were recruited from nephrology outpatient clinics. About 68.1% of participants were males. The mean age of the study population was 49.14 ± 13.63 years. There was a high proportion of illiteracy (36.3%) and unemployment (80.9%). KDCS showed a significant decline (P = 0.01) from CKD 3 to CKD 5D whereas MCS and PCS showed a nonsignificant decrease. There was no difference in KDCS, PCS, or MCS scores between patients treated by hemodialysis and CAPD. Illiteracy and unemployment were associated with significantly lower KDCS, PCS, and MCS scores. Age ≥50 years was associated with poor PCS (29.49 ± 8.20 vs. 34.17 ± 9.99; P < 0.001). Hemoglobin <10 g/dL was associated with poor KDCS (58.93 ± 13.09 vs. 65.55 ± 13.38; P < 0.001) and PCS (29.56 ± 8.13 vs. 33.37 ± 9.82; P < 0.001). The presence of comorbidities such as diabetes and hypertension had no impact on the composite scores. KDCS, MCS, or PCS scores did not vary among patients having high serum phosphorus (≥4.5 mg/dL), low albumin (<3.5 g/dL), and elevated parathyroid hormone (≥150 pg/ml). On multiple linear regression analysis, the predictors of KDCS were unemployment (P < 0.001) and illiteracy (P = 0.03). Unemployment (P < 0.001) and age (P < 0.001) were predictors of PCS whereas literacy level (P < 0.001) was predictive of MCS.

Clinical spectrum and outcomes of crescentic glomerulonephritis: A single center experience.

There is limited data on the etiology, clinical and histopathological spectrum and outcomes of crescentic glomerulonephritis (CrGN) in adult Indian population. This prospective study was done to evaluate the etiology, clinicohistological patterns and predictors of outcome of CrGN in South Indian population. All the patients received standard protocol based immunosuppression in addition to supportive care. Immune-complex glomerulonephritis (ICGN) was the most common etiology (n = 31; 77.5%) followed by pauci-immune glomerulonephritis (PauciGN; n = 8; 20%) and anti-glomerular basement membrane disease (n = 1; 2.5%). The most common etiology of ICGN was IgA nephropathy (n = 11; 27.5%) followed by lupus nephritis (n = 7; 17.5%) and post-infectious glomerulonephritis (PIGN) (n = 7; 17.5%). The patients with PauciGN were significantly older compared to those with ICGN (44.5 ± 15 years vs. 31.8 ± 11 years; P = 0.01). The patients with PauciGN presented with significantly higher serum creatinine (9.7 ± 4.4 vs. 6.6 ± 3.3 mg/dl; P = 0.03). The histopathologic parameters of ICGN and PauciGN were comparable except for a higher proportion of sclerosed glomeruli in ICGN. At the end of 3 months follow-up, only two patients went into complete remission (5.4%). Majority of the patients had end-stage renal failure (48.6%) and were dialysis dependent and seven patients (18.9%) expired. There was no signifi difference in the renal survival (10.9 ± 1.9 vs. 9.6 ± 3.3 months) or patient survival (17.5 ± 2.1 vs. 17.3 ± 4.3 months). The parameters associated with adverse outcomes at 3 months were hypertension (odds ratio [OR]: 0.58; confidence interval [CI]: 0.36-0.94), need for renal replacement therapy (OR: 0.19; CI: 0.04-0.9), serum creatinine at admission (P = 0.019), estimated glomerular filtration rate (P = 0.022) and percentage of fibrocellular crescents (P = 0.022).

Pattern and outcome of Cleistanthus collinus (Oduvanthalai) poisoning in a tertiary care teaching hospital in South India.

There is paucity of information on human studies about Cleistanthus collinus (Oduvanthalai) poisoning at global level. The present study was done to find out the pattern and outcomes with acute poisoning of this plant poison. Retrospective record based study was conducted among acute C. collinus (Oduvanthalai) poisoning cases admitted between January 2010 and December 2010 in a tertiary care teaching hospital in South India. A total of 51 cases were analyzed with 52.9% of them being females and 51% belonged to 21-40 years age group. Interpersonal conflict was the stressor for poisoning in 76% cases. Mortality rate was 17.6% with a median duration of 3.5 days from time of ingestion. Majority of the patients who died during hospitalization had ingested decoction (77.8%), and had neurological manifestations (77.8%), hypokalemia (77.8%), neutrophilia (66.7%), leucocyotosis (55.6%) and elevated blood urea (77.8%). It was found that lower potassium level, white blood cell and neutrophil count were significantly associated with mortality due to poisoning.