Leuprorelin 3.75mg powder and solvent for suspension for injection vials

Although leuprorelin acetate, a luteinizing hormone-releasing hormone agonist, has been approved based on short-term clinical trials conducted in Japan, its long-term efficacy on physical function remains unclear. We aimed to evaluate the long-term therapeutic efficacy of leuprorelin acetate using real-world clinical data through a self-controlled trend-shift analysis. The analysis included 91 genetically confirmed patients with spinal and bulbar muscular atrophy, with follow-up data collected before and after treatment initiation. The functional outcomes assessed included the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) and modified Norris scales, grip power, and serum creatinine levels. Leuprorelin acetate significantly slowed disease progression. For instance, the annual ALSFRS-R decline rate improved from approximately 0.5 points pre-treatment to 0.2 points post-treatment. The subgroup analysis supported the potential benefit of early intervention. These findings highlight the value of leveraging patient registries and post-marketing real-world data to evaluate treatment efficacy in slowly progressive diseases, such as SBMA, where traditional randomized controlled trials are often limited by insufficient statistical power to detect therapeutic efficacy. They also underscore the need for innovative methodologies to assess post-approval drug performance, paving the way for improved clinical outcomes for neurodegenerative diseases.

Abstract licence: CC BY-NC-ND

Background and clinical significanceRelugolix combination therapy (Relugolix CT) has emerged as an effective oral medical treatment for symptomatic uterine fibroids, offering an alternative to surgical interventions. While generally well tolerated, reports of adverse events beyond the common side effects are limited.Case presentationA 34-year-old nulliparous woman presented with abdominal pain and abnormal uterine bleeding. She had previously been diagnosed with a large FIGO Type 1-5 anterior wall fibroid and started on Relugolix CT as a bridge to surgery. At referral, speculum examination revealed a necrotic, malodorous, partially expelled mass protruding through the cervix. Surgical removal under spinal anaesthesia was performed, followed by resection of the intracavitary component. Histopathology confirmed leiomyoma with extensive necrosis. Postoperative imaging showed a residual fibroid, leading to discontinuation of Relugolix and initiation of leuprorelin acetate. The patient reported symptom resolution but was lost to follow-up after 3 months. This case highlights potential serious but under-recognized adverse effects associated with Relugolix CT, particularly in patients with large or intracavitary fibroids. Clinicians should maintain vigilance and ensure appropriate monitoring during treatment.ConclusionRelugolix CT represents a promising option in uterine fibroid management, but individualized patient evaluation and awareness of possible complications are essential to optimize safety and outcomes. The potential of this therapeutic approach warrants further investigation through randomized clinical trials, while real-world patient data are equally crucial to strengthen the evidence base and support broader clinical applicability.

Abstract licence: CC BY