Safety and efficacy of monthly high-dose vitamin D3 supplementation in children and adolescents with sickle cell disease.

Little is known about the impact of vitamin D supplementation on hand grip strength (HGS) and health-related quality of life (HRQoL) in children and adolescents with sickle cell disease (SCD). We aimed to evaluate the safety and efficacy of monthly high-dose vitamin D3 supplementation and its implications on bone mineral density (BMD), HGS, and HRQoL in patients with SCD and healthy controls. The study included 42 children with SCD and 42 healthy matched controls. The study participants were supplemented with high-dose monthly oral vitamin D3. Changes in the serum level of 25(OH) vitamin D3, maximum HGS, and BMD from baseline to 6 months were assessed, and the HRQoL questionnaire and Childhood Health Assessment Questionnaire (CHAQ) were used to evaluate the functional capacity. At baseline, SCD subjects had poorer growth status indicated by negative Z scores. Suboptimal BMD was detected by significantly lower Z score, and lower HGS and worse HRQL parameters were found compared to the controls (P < 0.001). Median 25(OH) vitamin D3 was significantly lower in SCD patients compared to controls (16.5 vs. 28 ng/mL, respectively (P < 0.001)). After 6 months of vitamin D supplementation, there was significant improvement in the DEXA Z-score (P < 0.001), limitation of physical health (P = 0.02), pain scores (P < 0.001), and CHAQ grades (P = 0.01) in SCD patients. A significant improvement in HGS (P < 0.001 and P = 0.005) as well as the CHAQ score (P < 0.001 and P = 0.003) was detected in the SCD group and controls, respectively. There were no reported clinical adverse events (AEs) or new concomitant medications (CMs) during the study duration, and safe levels of Ca and 25 (OH) D3 were observed at 3 and 6 months for both groups. There was a significant positive correlation between HGS and total physical score (r = 0.831, P < 0.001) and a negative correlation with CHAQ score (r = - 0.685, P < 0.001). We also detected a significant positive correlation between vitamin D levels at 6 months and HGS (r = 0.584, P < 0.001), pain score (r = 0.446, P < 0.001), and a negative correlation with CHAQ score (r = - 0.399, P < 0.001).   Conclusion: Monthly oral high-dose vitamin D supplementation was safe and effective in improving vitamin D levels, HGS, and HRQoL in SCD children and healthy subjects, and BMD scores in SCD patients. Further randomized controlled trials are warranted to assess an optimal dosing strategy and to investigate the impact on clinically significant outcomes in children and adolescents with SCD and their healthy counterparts.   Trial registration: ClinicalTrials.gov , identifier NCT06274203, date of registration: 23/02/2024, retrospectively registered. What is known: • Several studies have reported a high prevalence of vitamin D deficiency and suboptimal bone mineral density (BMD) in sickle cell disease (SCD) patients. • Musculoskeletal dysfunction is reported in SCD patients with a negative impact on physical activity and health-related quality of life (HRQL). • Little is known regarding the impact of vitamin D3 supplementation in children and adolescents with SCD. What is new: • We found that monthly oral high-dose vitamin D3 supplementation was safe, tolerated, and effective in improving serum vitamin D levels, HGS, BMD scores, and HRQL in SCD patients.

Damage accrual in Behçet disease: Behçet's Syndrome Overall Damage Index (BODI) versus Behçet's Disease Damage Index (BDI).

OBJECTIVES: To assess the validity of Behçet's Syndrome Overall Damage Index (BODI) and Behçet's Disease Damage Index (BDI) as tools for the detection of damage accrual in Behçet's patients compared to Vasculitis Damage Index (VDI). Also, to evaluate the correlation and the interclass correlation among the 3 indices to find out their consistency. METHOD: A prospective cohort study was carried out on 102 adult Behçet's disease (BD) patients who were diagnosed according to the International Study Group criteria for BD. Disease severity and organ damage were assessed for each patient by VDI, BDI and BODI at baseline and 1-year follow-up visits. Damage accrual for each index was defined when there was an increase of at least 1 point (∆ ≥ 1) among the baseline and the follow-up visits. RESULTS: Correlations among the 3 indices were significant, with (r = 0.835, P < 0.001) between VDI and BODI, (r = 0.835, P < 0.001) between VDI and BDI, and (r = 0.844, P < 0.001) between BODI and BDI scores. A highly significant positive correlation existed between the 3 indices and age and disease duration. In contrast, the correlation with the BD Current Activity Form was non-significant, which indicates good discriminative validity of the 3 indices. Neuropsychiatric and ocular systems showed a strong interclass correlation among the 3 indices. Regarding detecting damage accrual, BDI was more sensitive than BODI and showed more agreement with VDI. CONCLUSION: BD damage indices, VDI, BODI and BDI, had good convergent and discriminative validity for the assessment of BD damage. BDI had more sensitivity than BODI to the detection of damage accrual.

Utility of neutrophil/albumin ratio and C-reactive protein/albumin ratio as novel inflammatory markers in Behcet's disease.

INTRODUCTION AND OBJECTIVES: Behcet's disease (BD) is an autoimmune systemic inflammatory disease, and its exact pathogenesis is unknown. There are currently no specific tests to evaluate the disease activity of BD, making its management more difficult. This study aims to determine the neutrophil/albumin ratio (NAR) and C-reactive protein (CRP) to albumin ratio (CAR) and investigate their associations with clinical findings in patients with BD and to determine optimum cutoff levels of CAR and NAR. METHODS: The study included 75 consecutively recruited patients with BD with a mean age of 33.29±6.23 years and disease duration of 7.21±4.64 years, as well as 75 healthy participants. Clinical characteristics and laboratory data were obtained. Disease activity was assessed using the BD current activity form score (BDCAF). RESULTS: NAR and CAR were elevated in patients with BD compared with those of healthy controls (NAR: 1.08±0.34 vs. 0.607±0.06, CAR: 2.49±1.73 vs. 0.39±0.142; p<0.0001), as well as in active BD versus inactive BD. Both NAR and CAR were significantly correlated with CRP, active uveitis, and BDCAF score (p≤0.05). In patients with active BD, the cutoff value of NAR was >0.9744 (sensitivity: 89.74%, specificity: 80.56%), while that of CAR was >2.04 (sensitivity: 74.36%, specificity: 83.33%). CONCLUSIONS: NAR and CAR are both elevated in patients with BD and may contribute to its active state. NAR and CAR can be feasible and inexpensive markers for predicting BD activity.

Validity and reliability of the Arabic version of the Self-Efficacy for Managing Chronic Disease scale in rheumatoid arthritis patients.

BACKGROUND AND OBJECTIVES: Rheumatoid arthritis (RA) is an autoimmune inflammatory condition that causing disability and affection of patient's quality of life (QoL). Self-efficacy investigation helps us to detect the requirements of chronically affected patients and evaluation of self-care management programs. The aim of our study was to test validity and reliability of Self-Efficacy for Managing Chronic Disease (SEMCD-Arabic) in RA patients. METHOD: This study included 248 RA patients, carried out at Rheumatology and Rehabilitation Department. The SEMCD-Arabic Validity was assessed by correlating the SEMCD-Arabic scale with the validated Arabic version of the modified Health Assessment Questionnaire HAQ (MHAQ), the Arabic version of the Multidimensional Assessment of Fatigue (MAF) scale, and the Arabic version of Short Form 36 version 2 for quality of life (SF QoL). Internal consistency, test-retest reliability was assessed. RESULTS: Convergent validity was confirmed by a positive correlation between (physical, mental) component of SF QoL and SEMCD-Arabic (r = 0.918, r = 0.925) respectively, and negative correlation between MAF and SEMCD-Arabic (r = - 0.657) and MHAQ with SEMCD-Arabic (r = - 0.595). Discriminant validity confirmed by a significant negative correlation between visual analogue scale (VAS) for pain, disease activity scale (DAS28), Morning stiffness, patient health, physician health, age, duration, and SEMCD-Arabic (r = - 0.1-0.7) (P < 0.001). Test-retest reliability was estimated which revealed a high interclass correlation coefficient (ICC = 0.87-0.997) indicating excellent agreement and internal consistency is acceptable as the Cronbach's alpha value (0.660 to 0.78). CONCLUSION: The SEMCD-Arabic questionnaire can be used as a valid and reliable measure for assessment of patient's self-efficacy in RA. Key Points • The SEMCD-Arabic questionnaire has a statistically significant validity in correlation with different clinical manifestations MHAQ, SF QoL, and MAF. • The Arabic SEMCD is highly reliable with a Cronbach's alpha of 0.660 to 0.78.