Single-institute phase 2 study of thalidomide treatment for refractory or relapsed multiple myeloma: Prognostic factors and unique toxicity profile

Yutaka Hattori, Shinichiro Okamoto, Naoki Shimada, Tsunayuki Kakimoto, Kunihiko Morita, Yusuke Tanigawara, Yasuo Ikeda

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Abstract

We previously reported a pilot study of thalidomide monotherapy for Japanese patients with refractory or relapsed multiple myeloma. In the present work, we have extended this clinical trial to a single-institute phase 2 study with a larger number of patients and longer follow-up time. New information on the optimal dose and prognostic factors as well as the correlation of toxicities with treatment schedule was obtained. Fifteen of 56 (27%) patients achieved a partial response, including three cases with near-complete remission. Most patients suffered toxicities at a dose of 400 mg per day, but there was no clear dose-response relationship. Thus, a lower dose such as 200.mg per day or less is considered optimal. Multivariate analyses identified only lack of response to therapy as an adverse prognostic factor for progression-free survival. Chromosomal abnormality, C-reactive protein. >10 mg/L, and more than six previous courses of chemotherapy were significantly associated with shorter overall survival. Grade 3 or 4 neutropenia and thrombocytopenia were observed in 23 and 11% of patients, respectively. Grade 4 interstitial pneumonia and grade 5 pulmonary hypertension were observed; however, no patient suffered deep vein thrombosis, which has frequently been observed in other studies. Duration of therapy was closely related to the development of peripheral neuropathy. The efficacy and prognostic factors of this treatment were confirmed in long-term observation. However, special attention should be paid to toxicities such as hematological and pulmonary complications as well as peripheral neuropathy in long-term users.

Original languageEnglish
Pages (from-to)1243-1250
Number of pages8
JournalCancer Science
Volume99
Issue number6
DOIs
Publication statusPublished - 2008 Jun

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Thalidomide
Multiple Myeloma
Peripheral Nervous System Diseases
Therapeutics
Interstitial Lung Diseases
Neutropenia
Pulmonary Hypertension
Venous Thrombosis
Chromosome Aberrations
C-Reactive Protein
Disease-Free Survival
Appointments and Schedules
Multivariate Analysis
Observation
Clinical Trials
Drug Therapy
Lung
Survival

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

Cite this

Single-institute phase 2 study of thalidomide treatment for refractory or relapsed multiple myeloma : Prognostic factors and unique toxicity profile. / Hattori, Yutaka; Okamoto, Shinichiro; Shimada, Naoki; Kakimoto, Tsunayuki; Morita, Kunihiko; Tanigawara, Yusuke; Ikeda, Yasuo.

In: Cancer Science, Vol. 99, No. 6, 06.2008, p. 1243-1250.

Research output: Contribution to journalArticle

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