A multicenter phase 2 study of pomalidomide plus dexamethasone in patients with relapsed and refractory multiple myeloma: the Japanese MM-011 trial.
BACKGROUND
The immunomodulatory agent pomalidomide in combination with low-dose dexamethasone has demonstrated efficacy and safety for the treatment of relapsed and refractory multiple myeloma (RRMM) in phase 2 and 3 trials. However, these trials enrolled very few Asian patients.


METHODS
This phase 2 study investigated pomalidomide plus low-dose dexamethasone in 36 Japanese patients with RRMM after ≥2 prior therapies.


RESULTS
Patients enrolled in the study had a relatively high disease burden (81 % Durie-Salmon stage II or III) and were heavily pretreated (median, 6.5 prior antimyeloma regimens). The overall response rate was 42 % (1 patient with complete response and 14 with partial response), with an additional 44 % (16 patients) achieving stable disease (SD). Response rates in patients aged ≤65 years and >65 years were 47 and 35 %, respectively. None of the five patients with extramedullary disease achieved a response, with three of them maintaining SD of short duration. Median progression-free survival was 10.1 months after a 7.7-month median follow-up, and the median overall survival was not reached. The most frequent grade ≥3 adverse events (AEs) were neutropenia (64 %), anemia (42 %), and thrombocytopenia (31 %). The most frequent nonhematologic grade ≥3 AEs were pneumonia and decreased appetite (8 % each). Adverse events in patients aged >65 years were similar to those in patients aged ≤65 years, except for a higher rate of grade ≥3 pneumonia.


CONCLUSIONS
Collectively, the results of this study demonstrate that pomalidomide plus low-dose dexamethasone is an effective and safe treatment for Japanese patients with RRMM, although careful attention needs to be paid to serious infections.


BACKGROUND
Clinicaltrials.gov NCT02011113.
Department of Hematology and Oncology, Research Institute for Radiation Biology and Medicine, Hiroshima University, 1-2-3 Kasumi, Minami-Ku, Hiroshima 734-8553 Japan.
Tatsuo
T
Ichinohe
Department of Hematology and Oncology, Research Institute for Radiation Biology and Medicine, Hiroshima University, 1-2-3 Kasumi, Minami-Ku, Hiroshima 734-8553 Japan.
Yoshiaki
Y
Kuroda
Key University Hospital, 35 Shinanomachi, Shinjuku-Ku, Tokyo 160-8582 Japan.
Shinichiro
S
Okamoto
Kameda Medical Center, 929 Higashi-chou, Kamogawa, Chiba 296-8602 Japan.
Kosei
K
Matsue
Nagoya City University Hospital, 1 Kawasumi, Mizuho-chou, Mizuho-Ku, Nagoya 467-8601 Japan.
Shinsuke
S
Iida
Okayama Medical Center, 1711-1, Tamasu, Kita-Ku, Okayama 701-1154 Japan.
Kazutaka
K
Sunami
Mito Medical Center, 280 Sakuranosato, Ibarakimachi, Higashi-Ibaraki-Gun, Ibaraki 311-3193 Japan.
Takuya
T
Komeno
Japanese Red Cross Medical Center, 4-1-22 Hiroo, Shibuya-Ku, Tokyo 150-8935 Japan.
Kenshi
K
Suzuki
Tokai University Hospital, 143 Shimokasuya, Isehara, Kanagawa 259-1193 Japan.
Kiyoshi
K
Ando
University Hospital Kyoto Prefectural University of Medicine, Kawaramachi-Hirokoji, Kamigyo-Ku, Kyoto 602-8566 Japan.
Masafumi
M
Taniwaki
National Cancer Center Hospital, 5-1-1 Tsukiji, Chuo-Ku, Tokyo 104-0045 Japan.
Kensei
K
Tobinai
Niigata Cancer Center Hospital, 2-15-3 Kawagishimachi, Chuo-Ku, Niigata, 951-8133 Japan.
Takaaki
T
Chou
Osaka Red Cross Hospital, 5-30 Fudegasaki-cho, Tennouji-Ku, Osaka 543-8555 Japan.
Hitomi
H
Kaneko
Kyushu University Hospital, 3-1-1 Maidashi, Higashi-Ku, Fukuoka 812-8582 Japan.
Hiromi
H
Iwasaki
Celgene, 1-21-1 Toyama, Shinjuku-Ku, Tokyo 162-8655 Japan.
Chie
C
Uemura
Celgene, 1-21-1 Toyama, Shinjuku-Ku, Tokyo 162-8655 Japan.
Hiromi
H
Tamakoshi
Celgene Corporation, 86 Morris Avenue, Summit, NJ 07901 USA.
Mohamed H
MH
Zaki
Celgene Corporation, 86 Morris Avenue, Summit, NJ 07901 USA.
Thomas
T
Doerr
National Center for Global Health and Medicine Hospital, 15: 2-7-2 Marunouchi, Chiyoda-Ku, Tokyo 100-7010 Japan.
Shotaro
S
Hagiwara
Asian; Japan; Phase 2; Plasmacytoma; Pomalidomide; Relapsed/refractory multiple myeloma