{
  "abstract": "Background: The role of guideline-directed medical therapy for heart failure (HF) is unclear in transthyretin amyloid cardiomyopathy (ATTR-CM). Dapagliflozin has demonstrated beneficial effects across HF phenotypes, and retrospective studies suggest potential benefit in ATTR-CM; however, prospective data are lacking. This pilot study evaluated the effect and tolerability of dapagliflozin in patients with ATTR-CM to inform the design of a larger randomized trial. Methods: Participants were followed for 30 weeks over three study periods: 8 weeks baseline without study medication, 12 weeks intervention with dapagliflozin 10 mg once daily, and 10 weeks withdrawal without study medication. The study was open-label and nonrandomized. Vital signs and NT-proBNP were assessed every second week, while Kansas City Cardiomyopathy Questionnaire total symptom score (KCCQ-TSS) and 6-minute walking test (6MWT) were assessed every 4 weeks. Results were analyzed using linear mixed-effects models. Results: Seven of ten enrolled patients completed the trial. One participant discontinued because of genitourinary adverse effects, and two withdrew for reasons unrelated to the intervention. The estimated intervention-versus-baseline difference was −142 pg/mL (95% CI −324–41) for NT-proBNP, 24.3 m (95% CI −2.8 to 51.5) for 6MWT distance, and 0.4 points (95% CI −4.0 to 4.8) for KCCQ-TSS. Sitting SBP was 8.8 mmHg lower during intervention than during baseline (95% CI −13.2 to −4.4). No increase in hypotensive symptoms or cardiovascular adverse events was detected. Conclusions: Dapagliflozin was generally well tolerated in this pilot study of patients with ATTR-CM. The estimated differences in NT-proBNP, 6MWT distance, and KCCQ-TSS were imprecise and did not provide conclusive evidence of treatment effects. SBP was lower during treatment, without a detected increase in hypotensive symptoms. These exploratory findings support further evaluation in an adequately powered randomized trial. Trial registration: This study was registered with clinical trial number EudraCT no 2021-003674-32",
  "abstract_shown": true,
  "affiliations": [
    "Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden",
    "Department of Nursing, Umeå University, Umeå, Sweden",
    "Department of Diagnostics and Intervention, Umeå, Sweden",
    "Karolinska Institute, Department of clinical sciences, Stockholm, Sweden"
  ],
  "article_type": "Research Article",
  "arxiv": null,
  "author_total": 8,
  "authors": [
    {
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        0
      ],
      "name": "Erik Håkansson"
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    {
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        1
      ],
      "name": "Intissar Anan"
    },
    {
      "affs": [
        2
      ],
      "name": "Mattias Brunström"
    },
    {
      "affs": [
        3
      ],
      "name": "Karin Hellström-Ängerud"
    },
    {
      "name": "Per Lindqvist"
    },
    {
      "name": "Björn Pilebro"
    },
    {
      "name": "Jonas Wixner"
    },
    {
      "name": "Krister Lindmark"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/3q39wqemad400.bib",
  "cited_by": 0,
  "doi": "10.1371/journal.pone.0346065",
  "fulltext_url": null,
  "id": "3q39wqemad400",
  "issns": [
    "1932-6203"
  ],
  "landing_url": "https://doi.org/10.1371/journal.pone.0346065",
  "language": null,
  "licence": "CC-BY-4.0",
  "licence_label": "CC BY 4.0",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "openalex",
      "1312|Amyloidosis: Diagnosis, Treatment, Outcomes"
    ],
    [
      "plos",
      "/Biology and life sciences/Biochemistry/Hormones/Peptide hormones/Natriuretic peptide"
    ],
    [
      "plos",
      "/Medicine and health sciences/Cardiology/Cardiovascular medicine/Cardiovascular diseases/Cardiomyopathies"
    ],
    [
      "plos",
      "/Medicine and health sciences/Cardiology/Heart failure"
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      "/Medicine and health sciences/Endocrinology/Endocrine disorders/Diabetes mellitus"
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      "plos",
      "/Medicine and health sciences/Medical conditions/Cardiovascular diseases/Cardiomyopathies"
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      "plos",
      "/Medicine and health sciences/Medical conditions/Metabolic disorders/Diabetes mellitus"
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      "/Medicine and health sciences/Pharmaceutics/Drug therapy/Receptor antagonist therapy"
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    [
      "plos",
      "/Medicine and health sciences/Pharmacology/Drugs/Diuretics"
    ],
    [
      "plos",
      "/Medicine and health sciences/Vascular medicine/Blood pressure"
    ],
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      "plos",
      "/Research and analysis methods/Research design/Pilot studies"
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  "openalex": "W7220833676",
  "pdf_url": "https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0346065&type=printable",
  "pmcid": null,
  "pmid": null,
  "published": "2026-10-07",
  "publisher": "Public Library of Science",
  "ris": "https://research.draconitic.com/paper/3q39wqemad400.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T12:17:30Z",
      "native_id": "10.1371/journal.pone.0346065",
      "source": "plos",
      "url": "https://doi.org/10.1371/journal.pone.0346065"
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  "subjects": [
    {
      "field": "medicine",
      "origin": "plos:cardiovascular diseases",
      "subfield": "cardiovascular"
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    {
      "field": "medicine",
      "origin": "plos:endocrinology",
      "subfield": "endocrinology-metabolism"
    }
  ],
  "title": "Using dapagliflozin to reduce symptoms in transthyretin cardiac amyloidosis: An n-of-1 trial series pilot study",
  "title_text": "Using dapagliflozin to reduce symptoms in transthyretin cardiac amyloidosis: An n-of-1 trial series pilot study",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T12:17:30Z",
  "url": "https://research.draconitic.com/paper/3q39wqemad400",
  "venue": "PLOS One",
  "versions": []
}