1-Phenylalanine mustard (L-PAM) in the management of primary breast cancer. A report of early findings

N Engl J Med. 1975 Jan 16;292(3):117-22. doi: 10.1056/NEJM197501162920301.


Prolonged 1-phenylalanine mustard (L-PAM) administration as an adjuvant to mastectomy in the management of patients with primary breast cancer and pathologically positive axillary nodes was evaluated by a prospective, randomized, clinical trial. Treatment failures occurred in 22 per cent of 108 patients receiving placebo and 9.7 per cent of 103 women given L-PAM (p = 0.01). A statistically significant difference (p = 0.02) existed in favor of L-PAM relative to disease-free interval. In premenopausal women, the difference with respect to disease-free interval of treated and control groups was highly significant (p = 0.008). A treatment failure occurred in 30 per cent of premenopausal patients receiving placebo and 3 per cent of those treated with L-PAM (p = 0.008). Whereas a similar trend was observed in postmenopausal patients, the difference is not statistically significant. Thus, L-PAM has been demonstrated to be effective in the treatment of women with primary breast cancer, particularly those who are premenopausal. Results were achieved with minimal undesirable side effects.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Bone Marrow / drug effects
  • Bone Marrow Cells
  • Breast Neoplasms / drug therapy*
  • Breast Neoplasms / pathology
  • Clinical Trials as Topic
  • Drug Evaluation
  • Female
  • Humans
  • Lymph Nodes / pathology
  • Melphalan / administration & dosage
  • Melphalan / therapeutic use*
  • Melphalan / toxicity
  • Menopause
  • Middle Aged


  • Melphalan