title: Pomalidomide alone or in combination with dexamethasone in Japanese patients with refractory or relapsed and refractory multiple myeloma.
abstract: This phase 1, open-label, dose-escalation study investigated the tolerated dose (recommended dose), safety, efficacy, and pharmacokinetics of pomalidomide alone or pomalidomide plus low-dose dexamethasone in Japanese patients with refractory or relapsed and refractory multiple myeloma. Twelve patients were enrolled. Patients received pomalidomide 2 mg (Cohort 1) or 4 mg (Cohort 2) orally on day 1 and days 3-21 of a 28-day cycle. The tolerated dose of pomalidomide was determined to be 4 mg given on days 1-21 of a 28-day cycle. Efficacy outcomes with pomalidomide plus low-dose dexamethasone were consistent with those of previous studies. Responses (partial response or better) were achieved by three patients (25%; 1 [17%] in Cohort 1 and 2 [33%] in Cohort 2), and the median time to response was 6.4 months overall (9.0 months for Cohort 1 and 4.2 months for Cohort 2). The median progression-free survival was 5.5 months overall (5.1 months for Cohort 1 and not reached for Cohort 2). The most frequently occurring grade ≥3 adverse events were neutropenia (67%), anemia (25%), lymphopenia (25%), and pneumonia (25%), consistent with previous studies of pomalidomide plus low-dose dexamethasone in refractory or relapsed and refractory multiple myeloma. Further investigation of pomalidomide is recommended for Japanese patients with refractory or relapsed and refractory multiple myeloma. This study was registered with ClinicalTrials.gov (NCT01568294).
authors: Division of Hematology/Oncology, Department of Medicine, Kameda Medical Center, Kamogawa, Japan.
authors: Kosei
authors: K
authors: Matsue
authors: Center for Cellular and Molecular Medicine, Kyushu University Hospital, Fukuoka, Japan.
authors: Hiromi
authors: H
authors: Iwasaki
authors: Department of Internal Medicine, Niigata Cancer Center Hospital, Niigata, Japan.
authors: Takaaki
authors: T
authors: Chou
authors: Department of Hematology, National Cancer Center Hospital, Tokyo, Japan.
authors: Kensei
authors: K
authors: Tobinai
authors: Department of Hematology, National Hospital Organization, Okayama Medical Center, Okayama, Japan.
authors: Kazutaka
authors: K
authors: Sunami
authors: Department of Hematology/Oncology, Tokai University School of Medicine, Isehara, Japan.
authors: Yoshiaki
authors: Y
authors: Ogawa
authors: Celgene KK, Tokyo, Japan.
authors: Mari
authors: M
authors: Kurihara
authors: Celgene KK, Tokyo, Japan.
authors: Shuichi
authors: S
authors: Midorikawa
authors: Celgene Corporation, Summit, New Jersey, USA.
authors: Mohamed
authors: M
authors: Zaki
authors: Celgene Corporation, Summit, New Jersey, USA.
authors: Thomas
authors: T
authors: Doerr
authors: Department of Hematology and Oncology, Nagoya City University Graduate School of Medical Sciences, Nagoya, Japan.
authors: Shinsuke
authors: S
authors: Iida
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics; D044466:Asian People; D003907:Dexamethasone / Q000008:administration & dosage; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007564:Japan; D008297:Male; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy* / Q000401:mortality; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy / Q000401:mortality; D016879:Salvage Therapy; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000493:pharmacokinetics
chemical_list: D000970:Antineoplastic Agents; D013792:Thalidomide; D003907:Dexamethasone; C467566:pomalidomide
keywords: Clinical trial; dexamethasone; multiple myeloma; pharmacokinetics; pomalidomide