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Factor VIII (FVIII) replacement and emicizumab are effective at preventing bleeds in patients with hemophilia A (HA). Though benefits of emicizumab among inhibitor patients with HA (PwHA) are well established, more real-world evidence among non-inhibitor patients is needed. Using a United States healthcare claims database, we compared billed annualized bleed rates (ABRb) and the total cost of care (TCC) before and after switching from FVIII prophylaxis to emicizumab among non-inhibitor male PwHA. Bayesian inferences were used to assess the difference in ABRb and TCC per patient per year (PPPY) pre- versus post-prophylaxis switch. We included 101 non-inhibitor male PwHA aged between 3 and 63 years old who switched from FVIII prophylaxis to emicizumab prophylaxis in 2018 or 2019. The ABRb increased from 0.52 to 0.62 (p = 0.83) after switch. The posterior probability of the mean ABRb increasing after the switch was 75.54%. The TCC PPPY increased from $517,143 to $627,005 (p < 0.0001) after switch and the posterior probability of mean costs increasing after the switch was 99.80%. Personalization of care through the identification of the most appropriate therapy for each patient can optimize clinical and economic outcomes. Future real-world evidence research could help establish the value of prophylactic options in targeted populations such as the non-inhibitor male PwHA. [ABSTRACT FROM AUTHOR]

Objectives: The objectives of this study were to describe the characteristics and initial treatment patterns, healthcare resource use (HCRU), and costs of patients newly diagnosed with neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (PN).Methods: This was a retrospective study of individuals enrolled in the IBM MarketScan Multi-State Medicaid database from 1 October 2014 to 31 December 2017. Patients aged ≤18 years at the index date (first diagnosis of NF1 or PN, whichever occurred later) with at least 1 ICD-10-CM diagnosis code for both NF1 and PN were included. All-cause HCRU and the associated direct costs during the follow-up period were calculated per patient per year (PPPY) in 2018 USD.Results: A total of 383 patients were included with a mean follow-up of 448 days. Most patients were diagnosed by a specialist (63.5%). During the follow-up period, pain medications were used by 58.5% of patients, 25.1% were treated with chemotherapy, 7.1% received surgery for PN, 1.6% received MEK inhibitors, and 0.8% received radiation. Mean PPPY inpatient, outpatient, ER, pharmacy, and other visits were 1.4, 17.3, 1.6, 13.6, and 25.8, respectively. Mean ± SD (median) total PPPY healthcare costs were $17,275 ± $61,903 ($2889), with total medical costs of $14,628 ± $56,203 ($2334) and pharmacy costs of $2646 ± $13,303 ($26).Conclusions: This study showed that many pediatric patients newly diagnosed with NF1 and PN were initially treated with supportive care only, highlighting a substantial unmet medical need. This study also highlights the considerable economic burden among patients with NF1 and PN. [ABSTRACT FROM AUTHOR]

Background. Treatment of visceral leishmaniasis (VL) is far from satisfactory. There is an urgent need for a therapy that is efficacious, safe, affordable, and of short duration. Methods. A randomized open-label study was conducted to assess the efficacy and safety of 2 regimens of paromomycin administered intramuscularly. Group A received 11 mg/kg/day for 14 days (n = 217 ) and group B received 11 mg/kg/day for 21 days (n = 112) for the treatment of VL in India. Results. Mild grade injection site pain was the most common adverse event. There was no nephrotoxicity, but 4 patients in group A had to discontinue treatment because of grade 3 elevation of hepatic enzymes. Initial cure was observed in 91.2% and 96.4% of patients in group A and group B, respectively. Definitive cure at 6 months of follow up was seen in 82% of patients in group A and 92% of patients in group B by intention-to-treat analysis and in 84.3% of patients in group A and 92.8% of patients in group B by per protocol analysis. Conclusions. Although the cure rate in the group of patients who received the 14-day regimen was not optimal, the results with respect to initial cure were encouraging. Further studies that combine a short course of paromomycin with treatment with another antileishmanial agent are warranted. [ABSTRACT FROM AUTHOR]

Objective: To assess the effects of the COVID-19 pandemic on obstetric care and outcomes.Methods: A prospective observational single-center study was performed, including all antenatal and parturient women admitted from April to August, 2020. Data were collected regarding number of admissions, deliveries, antenatal visits, reason for inaccessibility of health care, and complications during pregnancy, and compared with data from the pre-COVID period of October 2019 to February 2020.Results: There was a reduction of 45.1% in institutional deliveries (P < 0.001), a percentage point increase of 7.2 in high-risk pregnancy, and 2.5-fold rise in admission to the intensive care unit of pregnant women during the pandemic. One-third of women had inadequate antenatal visits. The main reason for delayed health-seeking was lockdown and fear of contracting infection, resulting in 44.7% of pregnancies with complications. Thirty-two symptomatic women who tested positive for COVID-19 were managed at the center with good maternal and fetal outcomes.Conclusion: Although COVID-19 does not directly affect pregnancy outcomes, it has indirect adverse effects on maternal and child health. Emergency obstetric and antenatal care are essential services to be continued with awareness of people while maintaining social distancing and personal hygiene. [ABSTRACT FROM AUTHOR]