A multicenter phase 2 study of empirical low-dose liposomal amphotericin B in patients with refractory febrile neutropenia

Kotaro Miyao, Masashi Sawa, Mio Kurata, Ritsuro Suzuki, Reona Sakemura, Toshiyasu Sakai, Tomonori Kato, Satomi Sahashi, Natsuko Tsushita, Yukiyasu Ozawa, Motohiro Tsuzuki, Akio Kohno, Tatsuya Adachi, Keisuke Watanabe, Kaneyuki Ohbayashi, Yuichiro Inagaki, Yoshiko Atsuta, Nobuhiko Emi

Research output: Contribution to journalArticlepeer-review


Invasive fungal infection (IFI) is a major life-threatening problem encountered by patients with hematological malignancies receiving intensive chemotherapy. Empirical antifungal agents are therefore important. Despite the availability of antifungal agents for such situations, the optimal agents and administration methods remain unclear. We conducted a prospective phase 2 study of empirical 1 mg/kg/day liposomal amphotericin B (L-AMB) in 80 patients receiving intensive chemotherapy for hematological malignancies. All enrolled patients were high-risk and had recurrent prolonged febrile neutropenia despite having received broad-spectrum antibacterial therapy for at least 72 hours. Fifty-three patients (66.3 %) achieved the primary endpoint of successful treatment, thus exceeding the predefined threshold success rate. No patients developed IFI. The treatment completion rate was 73.8 %, and only two cases ceased treatment because of adverse events. The most frequent events were reversible electrolyte abnormalities. We consider low-dose L-AMB to provide comparable efficacy and improved safety and cost-effectiveness when compared with other empirical antifungal therapies. Additional large-scale randomized studies are needed to determine the clinical usefulness of L-AMB relative to other empirical antifungal therapies.

Original languageEnglish (US)
Pages (from-to)79-86
Number of pages8
JournalInternational journal of hematology
Issue number1
StatePublished - Jan 1 2017


  • Antifungal therapy
  • Empirical therapy
  • Febrile neutropenia
  • Hematological malignancy
  • Liposomal amphotericin B

ASJC Scopus subject areas

  • Hematology


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