Efficacy and Safety Analysis of Setmelanotide as a Novel Treatment for Hypothalamic Obesity

Background: Hypothalamic injury and impaired melanocortin-4 receptor (MC4R) pathway signaling, often a result of surgery or radiation for a benign tumor, may lead to hypothalamic obesity (HO). After injury, sudden weight gain and appetite changes unresponsive to existing therapies develop. Setmelanotide, an MC4R agonist, is approved for chronic weight management in patients with certain MC4R pathway-associated diseases. We report interim results of a Phase 2 study of setmelanotide in HO (NCT04725240). Methods: Patients aged 6-40 years with body mass index (BMI) >95th percentile (children 6 to <18 years) or >35 kg/m2 (adults >18 years) and HO caused by structural hypothalamic damage secondary to craniopharyngioma or other benign brain tumor, surgical resection, and/or chemotherapy were enrolled. The setmelanotide dose was initiated by age, with 2-4 weeks of titration to 3.0 mg once daily, followed by 12-14 weeks at the target dose. The primary endpoint was the proportion of patients achieving >5% BMI reduction at Week 16. A key secondary endpoint was the composite proportion of children with >0.2-point reduction in BMI Z score and adults with >5% weight loss. Hunger was assessed daily using a numerical rating scale, where 0 = not hungry at all and 10 = hungriest possible. Results: Eleven patients were included (baseline mean [SD;range] age, 14.6 [4.8;6-23] years and mean [SD] BMI, 38.7 [5.7] kg/m2). All patients (90% CI, 76.2%-100%) had >5% reduction in BMI (p<0.0001);81.8% (90% CI, 53.0%-96.7%) had >10% reduction (p<0.0001). Mean (range) change in BMI was -17.2% (-37.2%, -6.7%). Mean (SD) change in hunger score was -2.7 (2.6). Frequent adverse events included nausea (63.6%), vomiting (45.5%), diarrhea (36.4%), and COVID-19 (36.4%). Two patients discontinued because of adverse events. Conclusions: These early results warrant continued evaluation of setmelanotide in this population with a high unmet medical need and no approved therapies.

COVID-19 Care Clinic in a Medical Center: Lessons Learned.

OBJECTIVE: The purpose of this report is to describe the elements of a Covid-19 Care Clinic (CCC), patient demographics, and outcomes. METHODS: Descriptive statistics were used to describe demographics, clinical characteristics, and outcomes. This report is based on 4934 unique patients seen in the CCC who provided research authorization within a 10-month period of time (April 1, 2020-January 31, 2021). The CCC infection control processes consisted of a rooming process that mitigated SARS-COV-2 transmission, preparing examination rooms, using PPE by staff, in room lab drawing, and escorting services to minimize the time in clinic. RESULTS: Of the 4934 unique patients seen (age range newborn-102 years), 76.8% were tested for COVID-19. Of those tested, 11.8% were positive for SARS-CoV-2. Ninety-two percent of the patients with the reason for the visit documented had COVID-19 type symptoms. Cough, shortness of breath, and chest pain were the most common presenting symptom in those with COVID-19. At the time of the visit in the CCC, 5.8% of the patients were actively contagious. Thirty days after being seen in the CCC, 9.1% of the patients were seen in the emergency department (ED) and 0.2% died. During the 10-month period there were no known occupationally related COVID-19 infections. CONCLUSION: The COVID-19 Care Clinic provided face-to-face access for all ages with COVID-19 type symptoms. A minority of patients had COVID-19 who were seen in the clinic. The clinic provided an additional venue of care outside of the ED. The infectious control measures employed were highly effective in protecting the staff. Lessons learned allow for decentralization of COVID-19 symptom care to the primary care practices employing the infection control measures.

COVID Care Clinic: A Unique Way for Family Medicine to Care for the Community During the SARS-CoV-2 (COVID-19) Pandemic.

The COVID-19 pandemic has presented new challenges in how Primary Care clinicians care for community patients. Our organization quickly allocated 1 of our community clinic sites into a dedicated COVID Clinic caring for the COVID positive or any patient with COVID like symptoms to minimize contact with the well patients. A prerequisite for all patients to be seen in the COVID Care Clinic was a virtual visit staffed with Advanced Practice Providers that would further determine if the patient needed to seek emergency medical care or be seen in the COVID Clinic. From March 23, 2020 through May 15, 2020, 852 patients with COVID symptoms were seen in this clinic rather than the emergency department. This article describes a collaborative effort to care for a community during the COVID-19 pandemic. This unique setting allowed us to focus an appropriate level of care to a high risk population in a safe and effective manner in the ongoing effort to flatten the epidemiological curve.