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@ARTICLE{Gottschlich:304095,
      author       = {A. S. Gottschlich and J. Ernst and T. Milde$^*$ and B.
                      Gruhn},
      title        = {{C}omparative study of liposomal amphotericin {B},
                      posaconazole, and micafungin for primary antifungal
                      prophylaxis in pediatric patients with acute leukemia.},
      journal      = {Journal of cancer research and clinical oncology},
      volume       = {151},
      number       = {8},
      issn         = {0301-1585},
      address      = {Heidelberg},
      publisher    = {Springer},
      reportid     = {DKFZ-2025-01763},
      pages        = {235},
      year         = {2025},
      abstract     = {Invasive fungal diseases (IFDs) are a significant cause of
                      morbidity and mortality in pediatric patients with
                      hematologic malignancies including acute leukemia. Our study
                      aimed to compare the efficacy of liposomal amphotericin B
                      (L-AMB), posaconazole or micafungin as primary antifungal
                      prophylaxis (PAP) in pediatric patients with acute
                      leukemia.This retrospective observational study enrolled 95
                      pediatric patients with acute lymphoblastic leukemia (n =
                      70) or acute myeloid leukemia (n = 25), undergoing
                      chemotherapy, including those undergoing allogeneic
                      hematopoietic stem cell transplantation at the Department of
                      Pediatrics, Jena University Hospital, Jena, Germany. PAP
                      regimens included L-AMB (1 mg/kg/day or 3 mg/kg twice
                      weekly, intravenously), posaconazole (100-300 mg/day,
                      according to blood concentration, orally or intravenously)
                      and micafungin (1 mg/kg/day or 3 mg/kg twice weekly,
                      intravenously). Thirty-four patients $(35.8\%)$ received
                      L-AMB, 37 patients $(38.9\%)$ received posaconazole, and 24
                      patients $(25.3\%)$ received micafungin. Patients with a
                      history of IFD or concurrent or changing PAP were excluded.
                      The primary endpoint was the occurrence of breakthrough IFD,
                      while secondary endpoint included IFD-free survival.
                      Statistical analyses were performed using Kaplan-Meier
                      survival analysis, Gray's test and Cox regression to
                      evaluate IFD-free survival.The overall incidence of IFD was
                      $14.7\%$ (14 of 95 patients). IFD developed in 10 of 33
                      patients $(29.4\%)$ receiving L-AMB, in 4 of 38 $(10.8\%)$
                      patients receiving posaconazole and in none of the patients
                      receiving micafungin. IFD-free survival was $70.6\%$ in the
                      L-AMB group, $89.2\%$ in the posaconazole group and $100\%$
                      in the micafungin group (p = 0.005, log-rank test).
                      Significant differences were also observed in the cumulative
                      incidences of breakthrough IFDs (p = 0.006) assessed by
                      Gray's test. In multivariate Cox analysis, dichotomized
                      prophylaxis regimes (posaconazole or micafungin vs. L-AMB)
                      were independently associated with a reduced risk of IFD (HR
                      = 0.244; $95\%$ CI 0.076-0.777; p = 0.017). Age ≥ 10 years
                      predicted inferior IFD-free survival (HR = 3.665; $95\%$ CI
                      1.224-10.980; p = 0.020).We found a significant difference
                      in efficacy between the three antifungal prophylaxis
                      regimens. In our study, micafungin achieved the lowest IFD
                      breakthrough rate. However, multicenter clinical studies
                      would be needed to confirm the results.},
      keywords     = {Humans / Micafungin: administration $\&$ dosage / Male /
                      Female / Child / Antifungal Agents: therapeutic use /
                      Antifungal Agents: administration $\&$ dosage /
                      Retrospective Studies / Triazoles: administration $\&$
                      dosage / Triazoles: therapeutic use / Amphotericin B:
                      administration $\&$ dosage / Amphotericin B: therapeutic use
                      / Child, Preschool / Adolescent / Precursor Cell
                      Lymphoblastic Leukemia-Lymphoma: complications / Precursor
                      Cell Lymphoblastic Leukemia-Lymphoma: drug therapy /
                      Leukemia, Myeloid, Acute: complications / Leukemia, Myeloid,
                      Acute: drug therapy / Infant / Mycoses: prevention $\&$
                      control / Invasive Fungal Infections: prevention $\&$
                      control / Acute leukemia (Other) / Invasive fungal disease
                      (Other) / Liposomal amphotericin B (Other) / Micafungin
                      (Other) / Pediatric (Other) / Posaconazole (Other) / Primary
                      antifungal prophylaxis (Other) / Micafungin (NLM Chemicals)
                      / Antifungal Agents (NLM Chemicals) / posaconazole (NLM
                      Chemicals) / Triazoles (NLM Chemicals) / Amphotericin B (NLM
                      Chemicals) / liposomal amphotericin B (NLM Chemicals)},
      cin          = {B310},
      ddc          = {610},
      cid          = {I:(DE-He78)B310-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:40848057},
      doi          = {10.1007/s00432-025-06289-5},
      url          = {https://inrepo02.dkfz.de/record/304095},
}