{"doi":"10.3389/fneur.2024.1419791","title":"Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study","abstract":"Background and purpose: At 12 months in the phase 2 TOPAZ study, treatment with apitegromab was associated with both an improved motor function in patients with Type 2 or 3 spinal muscular atrophy (SMA) and with a favorable safety profile. This manuscript reports the extended efficacy and safety in the nonambulatory group of the TOPAZ study at 36 months. Methods: Patients who completed the primary study (NCT03921528) could enroll in an open-label extension, during which patients received apitegromab 20 mg/kg by intravenous infusion every 4 weeks. Patients were assessed periodically via the Hammersmith Functional Motor Scale-Expanded (HFMSE), Revised Upper Limb Module (RULM), World Health Organization (WHO) motor development milestones, Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT) Daily Activities and Mobility domains, and Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue questionnaire. Results: = 7 after scoliosis surgery). Caregiver-reported outcomes (PEDI-CAT and PROMIS Fatigue) showed improvements from baseline over 36 months. In addition, most patients (28/32) improved or maintained WHO motor milestones achieved at baseline. The most frequently reported treatment-emergent adverse events were pyrexia (48.6%), nasopharyngitis (45.7%), COVID-19 infection (40.0%), vomiting (40.0%), and upper respiratory tract infection (31.4%). Conclusion: The benefit of apitegromab treatment observed at 12 months was sustained at 36 months with no new safety findings.","journal":"Frontiers in Neurology","year":2024,"id":426087,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":18,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9531,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2024-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":739271,"name":"John W. Day","orcid":null,"position":1,"is_corresponding":false},{"id":236856,"name":"Darryl C. De Vivo","orcid":"0000-0001-8420-0795","position":2,"is_corresponding":false},{"id":1224896,"name":"Jena M. Krueger","orcid":"0009-0009-3471-3395","position":3,"is_corresponding":false},{"id":297632,"name":"Eugenio Mercuri","orcid":"0000-0002-9851-5365","position":4,"is_corresponding":false},{"id":456963,"name":"A. Nascimento","orcid":"0000-0003-4952-5732","position":5,"is_corresponding":false},{"id":944082,"name":"Amy Pasternak","orcid":"0000-0001-8841-2613","position":6,"is_corresponding":false},{"id":1224897,"name":"Elena Mazzone","orcid":"0000-0002-7857-5600","position":7,"is_corresponding":false},{"id":395880,"name":"Tina Duong","orcid":"0000-0003-3177-4722","position":8,"is_corresponding":false},{"id":608759,"name":"Guochen Song","orcid":"0009-0009-9502-1093","position":9,"is_corresponding":false},{"id":1225494,"name":"Jing L. Marantz","orcid":null,"position":10,"is_corresponding":false},{"id":1225495,"name":"Scott B. Baver","orcid":null,"position":11,"is_corresponding":false},{"id":1225496,"name":"Dongzi Yu","orcid":null,"position":12,"is_corresponding":false},{"id":1224898,"name":"Lan Liu","orcid":"0000-0002-6489-212X","position":13,"is_corresponding":false},{"id":254887,"name":"Basil T. Darras","orcid":"0000-0002-1536-8145","position":14,"is_corresponding":false},{"id":250711,"name":"Thomas O. Crawford","orcid":"0000-0003-0916-2797","position":0,"is_corresponding":true}],"reference_count":28,"raw_metadata":null,"created_at":"2026-07-19T01:58:37.924718Z","pmid":"39105058","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}