title: Long-term use of lenalidomide and low-dose dexamethasone in Chinese patients with relapsed/refractory multiple myeloma: MM-024 Extended Access Program.
abstract: BACKGROUND
  The efficacy and safety of lenalidomide plus low-dose dexamethasone (Rd) in Chinese patients with relapsed/refractory multiple myeloma (RRMM) was demonstrated in a phase 2, multicenter trial (MM-021). MM-024 was an Extended Access Program (EAP) that allowed responding patients in the MM-021 trial to continue to receive Rd, and to provide additional safety and efficacy data with longer follow-up.
  
  
  METHODS
  Chinese patients with RRMM who completed ≥ 1 year of Rd therapy in MM-021 and who remained progression-free under Rd entered the Treatment Phase of the MM-024 EAP, continuing Rd at the same dose and schedule. Patients in MM-021 who discontinued Rd treatment or progressed were allowed to enroll in the Safety Follow-Up Phase of the MM-024 EAP. Safety data, including the incidence of second primary malignancies (SPMs), were collected for ≥ 5 years from the time the last on-study patient enrolled in the MM-021 trial (primary end point). Efficacy outcomes (time to progression [TTP], progression-free survival [PFS], and overall survival [OS]) were secondary end points.
  
  
  RESULTS
  Median follow-up was 38.4 months for the safety population (n = 80) and 43.3 months for the treatment cohort (n = 41). In the safety population, Grade 3-4 adverse events (AEs) occurred in 60.0 % of patients; the most common grade 3-4 AEs were neutropenia (20.0%), decreased neutrophil count (13.8%), and anemia (11.3%). There was no evidence of cumulative toxicity, and no patients discontinued Rd due to AEs; 2 patients had SPMs. In the treatment cohort, median duration of response was 35.1 months, median TTP was 36.9 months, and median PFS was 36.0 months; median OS was not reached due to the low number of deaths (n = 5).
  
  
  CONCLUSIONS
  Long-term treatment with Rd has a predictable and manageable safety profile and provides sustained efficacy in Chinese patients with RRMM.
  
  
  BACKGROUND
  China State Food and Drug Administration (SFDA) registration (CTA reference numbers: 209L10808; 209L10809; 209L10810; and 209L10811) and ClinicalTrials.gov Identifier: NCT02348528. First received January 23, 2015; last updated November 12, 2015; last verified November 2015; study start date September 2012.
authors.affiliation: Guangdong General Hospital, Guangzhou, China. miyadu@hotmail.com.
authors.forename: Xin
authors.initials: X
authors.lastname: Du
authors.affiliation: The 1st Affiliated Hospital, Zhejiang University, Hangzhou, China. jiej0503@163.com.
authors.forename: Jie
authors.initials: J
authors.lastname: Jin
authors.affiliation: The 1st Affiliated Hospital, Zhejiang University, Hangzhou, China. caizhen1@sina.com.
authors.forename: Zhen
authors.initials: Z
authors.lastname: Cai
authors.affiliation: Xiangya Hospital of Central South University, Changsha, China. cnchenfp@hotmail.com.
authors.forename: Fangping
authors.initials: F
authors.lastname: Chen
authors.affiliation: Peking Union Medical College Hospital, Beijing, China. zhoudb@pumch.cn.
authors.forename: Dao-bin
authors.initials: DB
authors.lastname: Zhou
authors.affiliation: The 301 Hospital-Chinese PLA General Hospital, Beijing, China. liyu301@vip.163.com.
authors.forename: Li
authors.initials: L
authors.lastname: Yu
authors.affiliation: Peking University Third Hospital, Beijing, China. xykbysy@163.com.
authors.forename: Xiaoyan
authors.initials: X
authors.lastname: Ke
authors.affiliation: Shanghai 6th Hospital, Shanghai, China. lixiao3326@yahoo.com.cn.
authors.forename: Xiao
authors.initials: X
authors.lastname: Li
authors.affiliation: The 1st Affiliated Hospital of Soochow University, Suzhou, China. wudepei@medmail.com.cn.
authors.forename: Depei
authors.initials: D
authors.lastname: Wu
authors.affiliation: Nanfang Hospital of Southern Medicine University in Guangzhou, Guangzhou, China. mengfu@medmail.com.cn.
authors.forename: Fanyi
authors.initials: F
authors.lastname: Meng
authors.affiliation: Celgene Corporation, Summit, NJ, USA. ddemarco@celgene.com.
authors.forename: Dena
authors.initials: D
authors.lastname: DeMarco
authors.affiliation: Celgene Corporation, Summit, NJ, USA. jizhang@celgene.com.
authors.forename: Jingshan
authors.initials: J
authors.lastname: Zhang
authors.affiliation: Celgene Corporation, Summit, NJ, USA. jmei@celgene.com.
authors.forename: Jay
authors.initials: J
authors.lastname: Mei
authors.affiliation: Department of Hematology, Shanghai Changzheng Hospital, Shanghai, China. houjian@medmail.com.cn.
authors.forename: Jian
authors.initials: J
authors.lastname: Hou
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000971:Antineoplastic Combined Chemotherapy Protocols; D002681:China; D003907:Dexamethasone / Q000008:administration & dosage* / Q000009:adverse effects; D018572:Disease-Free Survival; D004305:Dose-Response Relationship, Drug; D019008:Drug Resistance, Neoplasm / Q000235:genetics; D064420:Drug-Related Side Effects and Adverse Reactions / Q000145:classification / Q000473:pathology*; D005260:Female; D006801:Humans; D000077269:Lenalidomide; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy* / Q000473:pathology; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000473:pathology; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*; D016896:Treatment Outcome
chemical_list: D013792:Thalidomide; D003907:Dexamethasone; D000077269:Lenalidomide
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