The FDA approval of Otarmeni, an AAV-based gene therapy for biallelic OTOF-associated sensorineural hearing loss, represents a conceptual shift in otology: from bypassing defective auditory physiology to restoring it. Unlike cochlear implantation, which circumvents damaged sensory structures, OTOF gene replacement targets a synaptic deficit in a structurally intact cochlea. Early clinical trial data are encouraging, with 75% of evaluable participants-children with molecularly confirmed biallelic OTOF variants enrolled in early-phase trials-achieving the primary hearing endpoint at 24 weeks. However, critical questions remain regarding long-term durability, the feasibility of redosing, and the surgical and logistical infrastructure required for intracochlear delivery. This commentary argues that otoferlin gene therapy initiates a new treatment hierarchy rather than rendering cochlear implantation obsolete: restore when biology permits, bypass when it does not. Realizing this potential will require advances in newborn genetic screening, workforce training, and equitable access to gene delivery.
J. Selvasingh, Justin R. Shinn· Otolaryngology Head & Neck S...· 0 citations
Summary Background Countries of the South Asian Association for Regional Cooperation (SAARC) are home to approximately 600 million children aged 0–14 years, representing a quarter of the global child population. We report incidence and mortality for childhood cancers among South Asia's eight nations to inform priority setting for research, programming for health services, and policy for childhood cancers. Methods Incidence and mortality of new childhood cancer diagnoses for all cancers combined and for selected leading cancer types were retrieved from GLOBOCAN 2022 database of Afghanistan, Bangladesh, Bhutan, India, Maldives, Nepal, Pakistan, and Sri Lanka. Age-standardized incidence rates (ASR) and mortality-to-incidence ratios (MIR) were calculated, together with statistical examination of cross-country variation in diagnostic structure and mortality patterns. Findings In SAARC nations, 37,716 new childhood cancer diagnoses and 17,698 deaths were estimated in 2022. India contributed 68.8% of subregional cases (25,939), and Pakistan contributed 20.8% (7841). Age-standardized incidence rates differed and were greatest in Sri Lanka (10.4/100,000) and Pakistan (10.1) and lowest in Bhutan (2.3) and Bangladesh (3.7). Age-standardized mortality rates ranged from 1.1/100,000 in Bhutan to 5.2/100,000 in Afghanistan (SAARC-wide ASMR 3.5/100,000). Boys represented 57% of cases overall, with the proportion varying across countries. Leukemia was the most common cancer in all countries (35–50%), followed by central nervous system (CNS) tumors (approximately 12% of cases). MIR ranged from 0.30 (Sri Lanka) to 0.57 (Afghanistan). Interpretation SAARC childhood cancer patterns reflect both population size and health system capacity, with a preponderance of leukemia and marked mortality-to-incidence ratio disparities across countries, especially for CNS cancers. There is a need to have subregional cooperation to address gaps in cancer registration, equitable access to specialty care, and continuity of treatment. Funding ECD is funded in part through the 10.13039/100000892Prostate Cancer Foundation Young Investigator Award. SM, SLW, CBJ, and ECD are funded in part through the Cancer Center Support Grant from the 10.13039/100000054National Cancer Institute (P30 CA008748).
T. Menon, J. Selvasingh, Taral K. Jella et al.· The Lancet Regional Health -...· 0 citations