{
  "abstract": "Introduction: Hemophilia A (HA) is a bleeding disorder caused by factor VIII (FVIII) deficiency, with severe forms associated with spontaneous bleeding and long-term musculoskeletal complications. Although FVIII prophylaxis is the standard of care, its frequent intravenous infusions and treatment burden limit adherence. Emicizumab, a subcutaneous (SC) bispecific monoclonal antibody, significantly reduces bleeding and improves quality of life. Recently implemented in the Brazilian public health system for children <7 years with HA (<2% FVIII), it represents a major shift in national care requiring structured real-world implementation. Objective: Describe the implementation of emicizumab and evaluate its impact in children with HA (<2% FVIII) at a Brazilian Hemophilia Treatment Center (HTC). Material and methods: Retrospective analysis of children with HA (FVIII activity <2%) who were aged <7 years at the time of approval by the Brazilian Ministry of Health. Eligible patients had no history of inhibitors. Clinical outcomes were compared between pre and post-emicizumab periods. Results: Emicizumab prophylaxis was implemented through a structured stepwise approach, according to the Brazilian protocol. Fifteen boys were included, with 12 of them having received prior factor prophylaxis, with a median follow-up of 3.1 months (0.6–4.1 months) after emicizumab maintenance therapy. Among them, 20% had previously required a central venous catheter for FVIII infusions. Before emicizumab, in patients receiving prior factor prophylaxis, the annualized bleeding rate (ABR) was 0 in 7 patients, 1 in 3, 2 in 1, and 6 in 1. In those without prior prophylaxis, median ABR was 4. After emicizumab maintenance initiation, no bleeding episodes were reported. Total hemarthrosis episodes decreased from 10 before emicizumab to 0 during follow-up. The median number of monthly venipunctures decreased from 12 to 2, accompanied by a substantial reduction in the number of medication vials dispensed to each family. Healthcare utilization decreased, with no HTC visits and only 1 emergency department visit (minor head trauma without bleeding). Among the 8 respondents, 4 reported pain or burning during administration and all families preferred the new treatment. Discussion: The results of this study demonstrate the efficacy and safety of emicizumab in children up to 7 years of age without inhibitors. The significant reduction in the ABR is consistent with data from pivotal studies. The lower utilization of healthcare services correlates directly with the decrease in severe bleeding episodes. The unanimous preference among families reflects the objective reduction in treatment burden and a perceived improvement in quality of life (based on the families report without measurement using a validated questionnaire). The benefits of SC therapy previously reported in the literature were confirmed in the present study: a reduced need for invasive procedures and, consequently, a lower risk of potential complications. Finally, the safety profile proved similar to those previously described, with most adverse events being mild, self-limiting and having no impact on the continuity of treatment. Conclusion: The initial experience with emicizumab prophylaxis validates its efficacy in bleeding prevention, confirms the favorable tolerability, while further reinforcing the improvements on their daily routine and overall well-being, associated with a reduced treatment burden.",
  "abstract_shown": true,
  "affiliations": [
    "Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (HCFMUSP), São Paulo, Brazil"
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 6,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Julia Loureiro SION"
    },
    {
      "affs": [
        0
      ],
      "name": "Rebeca Danielly da Fonseca FARIAS"
    },
    {
      "affs": [
        0
      ],
      "name": "Eliane Partite Nobre SANDOVAL"
    },
    {
      "affs": [
        0
      ],
      "name": "Erica OKAZAKI"
    },
    {
      "affs": [
        0
      ],
      "name": "Vanderson Geraldo ROCHA"
    },
    {
      "affs": [
        0
      ],
      "name": "Paula Ribeiro VILLAÇA"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/ejpx04bb903p0.bib",
  "cited_by": 0,
  "doi": "10.1016/j.htct.2026.108021",
  "fulltext_url": "http://www.sciencedirect.com/science/article/pii/S2531137926017682",
  "id": "ejpx04bb903p0",
  "issns": [
    "2531-1379",
    "2531-1387"
  ],
  "landing_url": "https://doi.org/10.1016/j.htct.2026.108021",
  "language": "en",
  "licence": "CC-BY",
  "licence_label": "CC BY",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "lcc",
      "RC633-647.5"
    ],
    [
      "openalex",
      "2720|Hemophilia Treatment and Research"
    ]
  ],
  "openalex": "W7223154222",
  "pdf_url": null,
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-23",
  "publisher": "Elsevier",
  "ris": "https://research.draconitic.com/paper/ejpx04bb903p0.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T13:43:20Z",
      "native_id": "89fc2a2e10b946a3abdd8557db199652",
      "source": "doaj",
      "url": "https://doi.org/10.1016/j.htct.2026.108021"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "lcc:RC633-647.5",
      "subfield": "endocrinology-metabolism"
    }
  ],
  "title": "IMPLEMENTING EMICIZUMAB PROPHYLAXIS IN A PUBLIC HEMOPHILIA CARE PROGRAM: EARLY EXPERIENCE IN BRAZILIAN CHILDREN WITH HEMOPHILIA A",
  "title_text": "IMPLEMENTING EMICIZUMAB PROPHYLAXIS IN A PUBLIC HEMOPHILIA CARE PROGRAM: EARLY EXPERIENCE IN BRAZILIAN CHILDREN WITH HEMOPHILIA A",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T13:43:20Z",
  "url": "https://research.draconitic.com/paper/ejpx04bb903p0",
  "venue": "Hematology, Transfusion and Cell Therapy",
  "versions": []
}