Gefitinib as first-line, compassionate use therapy in patients with advanced non-small-cell lung cancer

Lung Cancer. 2004 Mar;43(3):317-22. doi: 10.1016/j.lungcan.2003.10.010.


Purpose: To evaluate the efficacy of single-agent gefitinib (Iressa, ZD1839), an oral, epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor, as first-line compassionate use therapy for advanced non-small-cell Lung cancer (NSCLC).

Patients and methods: Twenty-five patients who were unfit or refused chemotherapy received oral gefitinib 250mg daily as first-line therapy for the treatment of recurrent or metastatic NSCLC in a compassionate use program at a single institution.

Results: Four of 22 evaluable patients (18%), two with adenocarcinomas and two with bronchioloalveolar carcinomas, had an objective response and five patients (23%) had stable disease. Duration of response or stable disease was 3.5-22+ months. Median time to progression was 2.2 months, median survival was 12.6 months and 1-year survival 52%. The partial response plus stable disease rate by Eastern Cooperative Oncology Group (ECOG) performance status (PS) was 4/5 for PS 0 patients; 3/6 for PS 1-2 patients; and 2/14 for PS 3-4 patients. The two patients with PS > 2 who derived benefit from gefitinib had PS 3 due to co-morbidities. Two patients discontinued therapy due to severe toxicities: one patient had severe liver dysfunction and hemorrhagic cystitis, and another patient developed diarrhea with hypotension. A correlation between rash and antitumor activity was noted. Of seven patients who received chemotherapy subsequent to gefitinib, one had a partial response, three had stable disease, two progressed, and one was non-evaluable for response.

Conclusion: We report encouraging response and survival results with gefitinib as first-line treatment in unselected patients with advanced NSCLC. Gefitinib monotherapy should undergo further evaluation as first-line therapy in advanced NSCLC.

MeSH terms

  • Adenocarcinoma / drug therapy
  • Adenocarcinoma / pathology
  • Adenocarcinoma, Bronchiolo-Alveolar / drug therapy
  • Adenocarcinoma, Bronchiolo-Alveolar / pathology
  • Administration, Oral
  • Adult
  • Aged
  • Aged, 80 and over
  • Antineoplastic Agents / therapeutic use*
  • Carcinoma, Non-Small-Cell Lung / drug therapy*
  • Carcinoma, Non-Small-Cell Lung / pathology
  • Carcinoma, Squamous Cell / drug therapy
  • Carcinoma, Squamous Cell / pathology
  • Disease Progression
  • Enzyme Inhibitors / therapeutic use*
  • Female
  • Gefitinib
  • Humans
  • Lung Neoplasms / drug therapy*
  • Lung Neoplasms / pathology
  • Male
  • Middle Aged
  • Neoplasm Recurrence, Local / drug therapy
  • Neoplasm Recurrence, Local / pathology
  • Protein-Tyrosine Kinases / antagonists & inhibitors
  • Quinazolines / therapeutic use*
  • Survival Rate


  • Antineoplastic Agents
  • Enzyme Inhibitors
  • Quinazolines
  • Protein-Tyrosine Kinases
  • Gefitinib