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FDA approves Anifrolumab as first new Lupus treatment in more than 10 years

Anifrolumab, an inhibitor of type 1 interferons, received approval from the Food and Drug Administration for the treatment of adults with moderate to severe systemic lupus erythematosus (SLE) who are receiving standard therapy.

Dose: 300 mg concentrate for solution for infusion.

Adverse Effects: The most common adverse reactions are upper respiratory tract infection, bronchitis, infusion-related reaction, and herpes zoster. The most common serious adverse reaction was herpes zoster.

Source: FDA

First drug for cold agglutinin disease

The US FDA approves first drug for Cold Agglutinin Disease. Sutimlimab-jome (Brand name: ENJAYMO) is a classical complement inhibitor indicated to decrease the need for red blood cell (RBC) transfusion due to hemolysis in adults with cold agglutinin disease.

Dose:  Injection: 1,100 mg/22 mL (50 mg/mL) in a single-dose vial

Adverse effects: Most common adverse reactions (incidence ≥10%) are respiratory tract infection, viral infection, diarrhea, dyspepsia, cough, arthralgia, arthritis, and
peripheral edema.

Source: FDA

First treatment for rare Uveal Melanoma

The drug is tebentafusp-tebn (Kimmtrak, from Immunocore Limited), which acts as a bispecific gp100 peptide-HLA-directed CD3 T-cell engager. It is the first of a novel class of T-cell receptor (TCR) bispecific immunotherapies under development at the company.

It is also the first and only therapy for the treatment of unresectable or metastatic uveal melanoma to be approved by the FDA, and it is the first therapy to demonstrate a survival benefit to patients with this disease. However, the indication specifies that the drug is for use only in patients who are HLA-A*02:01-positive.

Source: Immunocore

Pellagra case

History: A 14 yr female presented to ED with 6 weeks h/o well-defined darkening and thickening of the hands, feet, ankles, neck, and the upper trunk region including upper chest and back. She had similar lesions but milder form on lips and periorbital region. The affected area of the skin was said to get exacerbated after exposure or re-exposure to sunlight. She also complaint of recurrent episodes of passage of loose watery stools. What’s the diagnosis?

Pellagra is a disease caused by a lack of vitamin niacin (vitamin B3).

Symptoms include inflamed skin, diarrhea, dementia, and sores in the mouth. Areas of the skin exposed to either sunlight or friction are typically affected first. Over time affected skin may become darker, stiffen, peel, or bleed.

There are two main types of pellagra, primary and secondary.

Primary pellagra is due to a diet that does not contain enough niacin and tryptophan.

Secondary pellagra is due to a poor ability to use niacin within the diet. This can occur as a result of alcoholism, long-term diarrhea, carcinoid syndrome, Hartnup disease, and a number of medications such as isoniazid. Diagnosis is typically based on symptoms and may be assisted by urine testing.

Treatment is with either niacin or nicotinamide supplementation. Improvements typically begin within a couple of days. General improvements in diet.

Image by PubMed
Clinical Rounds by ClinicHours

Vitamin D supplementation did not decrease the risk of cancer or CVD

Vitamin D supplementation did not appear to influence the incidence of cancer or major cardiovascular disease (CVD) events in older adults who largely already had adequate vitamin D levels, according to a new randomized controlled study. In the cohort of nearly 2500 healthy individuals, the researchers found no differences in cancer or CVD incidence over 5 years between the groups randomly assigned to vitamin D supplementation and to placebo.

Source: The American Journal of Clinical Nutrition

Putty kidney case

History: A 46-year-old woman presented with symptoms of urgency, frequency and urge-incontinence for 2 years with h/o of fever. Her urine was acidic with few pus cells, but sterile on culture. Other laboratory tests were normal. Ultrasonography suggested hydronephrotic right kidney with internal echoes. X-ray KUB show multiple lobulated calcifications in the right renal region conglomerated into the shape of the kidney.

Extensive parenchymal calcification in a non functioning kidney forming its cast is called a ‘putty kidney’ which is a characteristic of end-stage renal TB.

Symptoms: Persistent cystitis, unresponsive to antibiotics, ↑urinary frequency, dysuria, loin discomfort, malaise, fever, weight loss, ulcer.

Diagnosis: USG, Xray KUB, CECT scan

Treatment: ATT

Image by: BMJ

FDA approves Cabotegravir ER Injectable for HIV PrEP

The FDA issued approval for extended-release injectable cabotegravir (Apretude, CAB-LA) for use in at-risk adults and adolescents weighing at least 35 kilograms (77 pounds) for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV.

The priority review approval was based on phase 2b-3 clinical trial data submitted to the agency this past August, after one of the studies was stopped early because of encouraging efficacy results of the first pre-planned interim endpoint analysis.

Apretude is given first as two initiation injections administered one month apart, and then every two months thereafter. Patients can either start their treatment with Apretude or take oral cabotegravir (Vocabria) for four weeks to assess how well they tolerate the drug.

Source: FDA.gov

Tinea Barbae case

History: A 45-year-old farmer came to dermatology OPD with C/O nonitchy red lumpy areas, pustules, and crusting around the hairs that can be pulled out easily. What’s the diagnosis?

Tinea barbae is a superficial dermatophyte fungal infection that is limited to the bearded areas of the face and neck. The most common causes are Trichophyton mentagrophytes and T. verrucosum.

Signs and symptoms

  • Pimple or blister
  • Swelling and redness around infected area
  • Red and lumpy skin
  • Crusting around hairs in infected areas and effortless pulling of hair.

Diagnosis:

  • Direct microscopic examination
  • Culture on Sabouraud agar

Treatment: Antifungals

  • Topical antifungals
  • Itraconazole can be recommended as one-pulse therapy at a dosage of 400 mg/d divided into 2 doses for 1 week.
Image by DermNet NZ

FDA approves Efgartigimod for Myasthenia Gravis

The US FDA has approved efgartigimod (Brand name: Vygart) a first-in-class, targeted therapy for adults with generalized myasthenia gravis (gMG) who test positive for the antiacetylcholine receptor (AChR) antibody.

Efgartigimod is an antibody fragment designed to reduce pathogenic IgG antibodies and block the IgG recycling process in patients with gMG. The novel agent binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels.

The most common side effects associated with the use of Vyvgart include respiratory tract infections, headache, and urinary tract infections. As Vyvgart causes a reduction in IgG levels, the risk of infections may increase. Hypersensitivity reactions such as eyelid swelling, shortness of breath, and rash have occurred. If a hypersensitivity reaction occurs, discontinue the infusion and institute appropriate therapy.

Source: FDA

Abrocitinib approved for Atopic Dermatitis in Europe

The oral Janus kinase 1 inhibitor abrocitinib has been approved in Europe for the treatment of moderate to severe atopic dermatitis (AD) in adults, who are candidates for systemic therapy. Approval by the European Commission was based on the results of studies that include four phase 3 clinical trials (JADE MONO-1, JADE-MONO-2, JADE COMPARE, JADE REGIMEN) and an ongoing open-label extension study (JADE EXTEND) in over 2,800 patients.

Source: Mdedge

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