Phase I study of inhaled doxorubicin for patients with metastatic tumors to the lungs

Gregory A. Otterson, Miguel A. Villalona-Calero, Sunil Sharma, Mark G. Kris, Anthony Imondi, Mirjam Gerber, Dorothy A. White, Mark J. Ratain, Joan H. Schiller, Alan Sandler, Michael Kraut, Sridhar Mani, John R. Murren

Research output: Contribution to journalArticlepeer-review

111 Scopus citations


Purpose: To evaluate the toxicity profile of inhalational doxorubicin in patients with malignant disease in the lung. Experimental Design: The OncoMyst Model CDD-2a inhalation device aerosolizes compounds to particles of 2 to 3 μm and prevents exhaled aerosol from escaping into the environment. Deposition efficiency of inhaled Technetium 99m was used to predict deposition of doxorubicin and calculate dose. Treatment was repeated every 3 weeks. No more than moderate pulmonary dysfunction was permitted (forced expiratory volume in 1 s, forced vital capacity, and diffusing capacity for carbon monoxide, all >50% predicted; resting SaO2 >90%). Results: Fifty-three patients were enrolled at 13 dose levels ranging from 0.4 to 9.4 mg/m 2. The most common histologic diagnoses were sarcoma (n = 19) and non-small cell lung cancer (n = 16). Dose-limiting toxicity (DLT) was observed at the 9.4 mg/m2 dose level when two of four patients experienced pulmonary DLT. Of 11 patients treated at the 7.5 mg/m2 dose level, only one showed DLT consisting of a decline in forced vital capacity of >20% from baseline. No significant systemic drug-related toxicity was observed. Several patients experienced declines in pulmonary function test variables, which were attributed to progressive disease. Observed activity included a partial response in a patient with metastatic soft tissue sarcoma previously treated with i.v. doxorubicin and ifosfamide. Conclusions: Inhaled doxorubicin is safe up to a dose of 7.5 mg/m2 every 3 weeks in patients with cancer who had normal to moderately impaired pulmonary function.

Original languageEnglish (US)
Pages (from-to)1246-1252
Number of pages7
JournalClinical Cancer Research
Issue number4
StatePublished - Feb 15 2007

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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