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FDA approves WAYRILZ (Rilzabrutinib) for chronic ITP in adults

The U.S. FDA approved WAYRILZ (rilzabrutinib) tablets for adults with persistent or chronic immune thrombocytopenia (ITP) who have not responded to previous treatments.

MOA: kinase inhibitor

Dosage: 400 mg BD PO

Key Safety Precautions:

  • Infections: Monitor closely
  • Liver toxicity: Check bilirubin and liver enzymes
  • Pregnancy: May cause fetal harm; use contraception
  • Drug interactions: Avoid CYP3A inhibitors/inducers, PPIs; take 2 hours before antacids or H2 blockers
  • Organ impairment: Not for moderate/severe liver or severe kidney disease

Side Effects: Most common: diarrhea, nausea, headache, abdominal pain, and COVID-19 infection.

Reference: FDA
Clinical Inshorts  by ClinicHours

FDA approves first generic Liraglutide for obesity treatment

The U.S. FDA approved Teva Pharmaceuticals’ generic version of liraglutide (Saxenda®), the first generic GLP-1 receptor agonist for chronic weight management.

Indication: It is indicated for adults with obesity (BMI ≥30) or overweight (BMI ≥27 with comorbidity) and for adolescents aged 12–17 years with obesity.

Dose: 0.6 mg once daily SC, titrated weekly to a maintenance dose of 3 mg SC daily.

Mechanism: Liraglutide mimics GLP-1, enhancing insulin secretion, slowing gastric emptying, promoting satiety, and reducing appetite.

Common side effects: nausea, vomiting, diarrhea, headache, and injection-site reactions. Serious risks include pancreatitis, gallbladder disease, and thyroid C-cell tumors.

This approval is expected to lower treatment costs, compared to the branded Saxenda (~$1,350/month), and broaden access to effective obesity therapy.

Reference: FDA.gov
Clinical Inshorts  by ClinicHours

Atezolizumab + Bevacizumab leads in survival for advanced HCC

A new network meta-analysis of nine phase 3 trials (n=6425) compared first-line treatments for advanced hepatocellular carcinoma (HCC), focusing on overall survival (OS) and health-related quality of life (HR-QoL).

Researchers evaluated immune checkpoint inhibitor (ICI) combinations, ICI monotherapy, and tyrosine kinase inhibitors, with most trials using sorafenib as the control. Time to deterioration was measured across six QoL domains: global health, physical function, fatigue, jaundice, pain, and abdominal swelling. Rankings were based on SUCRA scores and hazard ratios.

Key Findings:

  • Atezolizumab + Bevacizumab ranked best across four QoL domains—global health/QoL (SUCRA 0.85; HR 0.63), abdominal swelling (0.950; HR 0.57), jaundice (0.895; HR 0.77), and pain (0.861; HR 0.65).

  • It also outperformed all regimens when OS was integrated with HR-QoL.

  • Sintilimab + IBI305 offered the best OS probability (SUCRA 0.892) and ranked well in pain, fatigue, and abdominal swelling.

  • Tislelizumab preserved physical functioning and fatigue, making it an alternative for QoL-focused patients.

  • Sorafenib and lenvatinib monotherapy consistently ranked lowest.

Takeaway: Atezolizumab plus bevacizumab remains the strongest first-line therapy for advanced HCC when balancing both survival and quality of life, while other ICIs may provide niche QoL advantages.

Clinical Inshorts by ClinicHours

FDA approves Papzimeos (zopapogene imadenovec-drba) for recurrent respiratory papillomatosis

Papzimeos is the first and only FDA-approved therapy for the treatment of adults with RRP.

Introduction: PAPZIMEOS™ is a non-replicating adenoviral vector-based immunotherapy indicated for the treatment of adults with recurrent respiratory papillomatosis.

Dosage: 5×10¹¹ particle units (PU) per injection, given SC 4 times over 12 weeks.

Usage: Prior to first dose, surgical debulking of visible papillomas is recommended to minimize residual disease. Supplied as a single-dose vial containing 5×10¹¹ PU in 1 mL suspension.

Contraindications: None.

Warnings & Precautions: Injection-site reactions Monitor patients for at least 30 minutes after Rx. Thrombotic events.

Adverse reactions: Most common (≥5%): injection-site reactions, fatigue, chills, fever, muscle pain, and nausea.

Reference: FDA
Clinical Inshorts by ClinicHours

2025 AHA hypertension guidelines: Key updates for clinical practice

Blood Pressure Categories & Treatment Targets

The guideline retains the 2017 definitions of blood pressure categories:

  • Normal: < 120/80 mm Hg
  • Elevated: 120–129 / < 80 mm Hg
  • Stage 1 Hypertension: 130–139 or 80–89 mm Hg
  • Stage 2 Hypertension: ≥ 140 or ≥ 90 mm Hg

The treatment goal for all adults remains < 130/80 mm Hg.

Risk Assessment & Personalized Care

The new PREVENT™ risk calculator replaces the older pooled cohort equations. It estimates both 10- and 30-year cardiovascular risk and integrates cardiovascular, kidney, metabolic, and social health factors to personalize interventions.

Prevention, Lifestyle & Brain Health

The guideline emphasizes prevention of heart disease, stroke, kidney disease, and cognitive decline.
Lifestyle recommendations include:

  • Sodium intake < 2,300 mg/day (ideal < 1,500 mg)
  • DASH-style or heart-healthy diet
  • 75–150 minutes/week of aerobic and/or resistance activity
  • ≥ 5% weight loss if overweight
  • Stress management with meditation, breathing exercises, or yoga
  • Alcohol limitation: maximum 2 drinks/day (men), 1 (women)

Diagnostics & Laboratory Evaluation

  • Urine albumin-to-creatinine ratio is now recommended for all hypertensive patients to assess kidney health.
  • Plasma aldosterone-to-renin ratio screening is expanded to detect primary aldosteronism, especially in resistant hypertension or sleep apnea.

Medication Strategy

  • Stage 2 hypertension: Start with two medications, preferably as a single-pill combination.
  • Recommended classes: ACE inhibitors, ARBs, long-acting dihydropyridine CCBs, and thiazide-type diuretics.
  • In patients with overweight or obesity, GLP-1 receptor agonists may be considered as an adjunct.

Special Considerations: Hypertension in Pregnancy

  • Initiate antihypertensive therapy when BP reaches ≥ 140/90 mm Hg.
  • Postpartum women with pregnancy-associated hypertension should have annual BP checks.

Low-dose aspirin (81 mg/day) may be considered to reduce the risk of preeclampsia.

Reference: AHA
Clinical Inshorts by ClinicHours

Ruxolitinib outperforms best available therapy in chronic GVHD

A phase 3, randomized, open-label trial of 329 patients (≥12 years) with steroid-refractory/dependent chronic graft-vs-host disease (SR/D-cGVHD) found that ruxolitinib significantly extended median failure-free survival (FFS) to 38.4 months versus 5.7 months with best available therapy (BAT) (HR 0.36).

At 36 months, 59.6% of ruxolitinib-treated patients maintained response vs 26.7% with BAT. Among BAT patients who crossed over, the week-24 overall response rate was 50%, with best overall response reaching 81.4%. Nonrelapse mortality was slightly lower with ruxolitinib (17.8% vs 22.0%).

The treatment showed sustained efficacy and manageable safety over 3 years, though some long-term endpoints (like quality of life) couldn’t be fully assessed due to dropouts.

Study led by Robert Zeiser, MD; funded by Novartis and Incyte; published June 25 in Journal of Clinical Oncology.

Clinical Inshorts by ClinicHours

Enlarged thymus at 2 months linked to higher risk of Atopic Dermatitis

Infants with a larger thymus at 2 months of age had a significantly higher risk—over sixfold—for developing atopic dermatitis (AD) by age 2. The risk remained high—over fivefold—for early-onset AD before 6 months.

Study Overview: A prospective study of 300 full-term infants at Rigshospitalet, Copenhagen (2017–2019) tracked thymus size via ultrasound at birth, 2 months, and 12 months. AD severity was assessed using the Eczema Area and Severity Index (EASI). Out of 300, 290 infants completed the 2-year follow-up.

Results:

  • AD prevalence by age 2: 34.1%

  • High thymic index at 2 months:

    • 6.3x higher AD risk by age 2 (aHR: 6.32; P < .001)

    • 5.4x higher risk for early-onset AD (<6 months) (aHR: 5.35; P < .001)

  • Moderate correlation between thymic index and EASI score at 2 months (r = 0.39); no correlation at birth or 12 months.

Clinical Insight: The association held even after adjusting for filaggrin (FLG) gene mutations and family history, indicating thymic size may independently predict AD risk.

Source: Jacob P. Thyssen, MD, PhD, University of Copenhagen; study published online July 29 in Allergy.

Limitations: Findings may be limited by potential bias from AD overrepresentation, variability in ultrasound quality, and reliance on thymus size alone.

Funding & Disclosures: Supported by multiple foundations including LEO, Lundbeck, Novo Nordisk, and others. Some authors reported financial ties or employment with sponsors.

Clinical Inshorts by ClinicHours

ASTRO guidelines incorporate the 2021 WHO glioma reclassification

Glioblastoma (GBM), now classified as CNS WHO grade 4 adult-type diffuse glioma, is the most aggressive and common malignant brain tumor in adults. Despite advancements in surgery, radiation therapy (RT), and chemotherapy (notably temozolomide), prognosis remains poor—median survival is 15–17 months and <10% survive 5 years.

Historically, GBM classification relied solely on histology, but the 2021 WHO CNS classification incorporates molecular markers for more accurate diagnosis and prognosis. Key molecular features defining WHO grade 4 diffuse glioma include:

  • IDH-wildtype astrocytoma with:

    1. EGFR amplification

    2. Gain of chromosome 7/loss of chromosome 10

    3. TERT promoter mutation

  • CDKN2A/B homozygous deletion in IDH-mutant gliomas

These markers can now reclassify some tumors previously labeled as grade 2 or 3 into grade 4, even without necrosis or vascular proliferation.

Importantly, only IDH-wildtype tumors are now classified as GBM, whereas IDH-mutant grade 4 tumors are termed astrocytoma, IDH-mutant, WHO grade 4.

This updated ASTRO guideline replaces the 2016 version, aligning with modern molecular classifications and addressing disparities in care to guide future research and equitable treatment.

Clinical Inshorts by ClinicHours

Semaglutide emerges as a potential treatment for MASH

Semaglutide, a glucagon-like peptide-1 receptor agonist, is a candidate for the treatment of metabolic dysfunction–associated steatohepatitis (MASH).

Methods: In this phase 3 trial, 1197 patients with biopsy-confirmed MASH and liver fibrosis (stage 2 or 3) were randomly assigned to weekly semaglutide 2.4 mg or placebo. Interim results at 72 weeks from the first 800 patients are reported. Primary goals were resolution of steatohepatitis without worsening fibrosis and improvement in fibrosis without worsening steatohepatitis.

Results: Semaglutide led to steatohepatitis resolution without fibrosis worsening in 62.9% of patients vs. 34.3% with placebo. Fibrosis improved without worsening steatohepatitis in 36.8% vs. 22.4%. Both outcomes occurred in 32.7% vs. 16.1%. Weight loss was greater with semaglutide (−10.5% vs. −2.0%). GI side effects were more common with semaglutide; pain scores were similar.

Conclusions: Semaglutide 2.4 mg weekly significantly improved liver histology in MASH patients with fibrosis.

Reference: NEJM
Clinical Inshorts by ClinicHours

New guidelines prioritize Iron over Dopamine in Restless Legs Syndrome

Restless Legs Syndrome (RLS) affects 8% of the population, mainly women. It causes an overwhelming urge to move the legs, especially in the evening or during rest, often with tingling or electric sensations. Movement temporarily relieves symptoms.

Previously, dopamine agonists (e.g., pramipexole, rotigotine, ropinirole) were first-line treatment. However, the American Academy of Sleep Medicine (AASM) now recommends against them due to augmentation syndrome—a worsening of symptoms with long-term use. France still permits their use in severe cases, per 2019 guidelines.

At a recent neurology conference in Montpellier, Dr. Sofiène Chenini emphasized non-dopaminergic options and warned against overuse.

Updated First-Line Therapies:

  • IV iron supplementation (when ferritin <75 µg/L) is now first-line.
  • Antiepileptics (gabapentin, pregabalin) are preferred for mild-to-moderate RLS or in cases with insomnia.
  • Lifestyle changes—avoiding caffeine, alcohol, smoking; regular sleep habits; and daytime exercise—can help mild cases.

If oral iron fails, IV options like ferric carboxymaltose or ferric hydroxide-sucrose are recommended. Mild opioids(e.g., codeine, tramadol) may be added if symptoms persist.

Diagnosis Criteria (SFRMS 2016)

  1. Urge to move legs with unpleasant sensations
  2. Worsens at rest
  3. Relieved by movement
  4. Worse at night
  5. No other underlying cause

RLS is linked to brain iron deficiency—MRI studies suggest impaired iron transport and dopamine/glutamate overactivity. Contributing factors include aging, certain medications (especially serotonergic antidepressants and antihistamines), and genetic predisposition.

Alternative antidepressants like venlafaxine or duloxetine (shorter half-life) are preferred if needed.

Misuse Drives Change

A U.S. registry of 670,000 RLS patients revealed 60% received dopamine agonists, and 20% exceeded recommended doses, especially among neurologists. Overprescription—often modeled on Parkinson’s protocols—prompted AASM’s guideline update.

Clinical Inshorts by ClinicHours
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