自体造血干细胞移植可使中国高危多发性骨髓瘤患者获益更多

目的 评价自体造血干细胞移植（ASCT）对初治多发性骨髓瘤（MM）的疗效.方法 选取诱导治疗后获得部分缓解（PR）及以上疗效的42例初治MM患者进行ASCT,中位随访34.2个月后,观察患者的疗效、无进展生存（PFS）和总生存（OS）.选择同时期的49例诱导治疗后获得PR及以[上疗效的初治MM患者,进入非移植组（non-ASCT）,经巩固维持治疗后观察疗效、PFS和OS,比较两组的差异,分析ASCT在MM中的作用.结果 与non-ASCT组相比,ASCT可明显延长患者的OS（未达到/37.2个月,P=O.000）,而且对PFS也有延长趋势（33.9个月/39.8个月,P=0.133）.ASCT可明显改善DSⅢ期（P=0.009）和ISSⅢ期（P=0.049）患者PFS,但对DSⅠ /Ⅱ期和ISS Ⅰ/Ⅱ期患者的PFS改善不明显.ASCT可明显改善诱导治疗后获得CR患者的PFS（P=0.016）,对非常好的PR （VGPR） /PR患者的PFS改善不明显;不同年龄患者OS均明显改善（≤55岁,P=0.039;＞ 55岁,P=0.000）.ASCT可明显改善不同ISS分期患者的OS（Ⅰ/Ⅱ期,P=0.003;Ⅲ期,P=0.012）,对DSⅢ期患者的OS也有明显改善作用（P=0.000）,但对DSⅠ/Ⅱ期患者OS的作用不明显（P=0.446）.诱导治疗后获得CR和VGPR/PR的患者进行移植后,OS可进一步得到改善（CR,P=0.004; VGPR/PR,P=0.004）.结论 ASCT可明显改善初治MM患者的生存,使高龄和分期预后不良的MM患者获益更多.     

GP和FP治疗转移性鼻咽癌的疗效观察

目的：观察吉西他滨＋顺铂（GP）方案和氟尿嘧啶＋顺铂（FP）方案治疗晚期复发转移性鼻咽癌的疗效及不良反应。方法：选择放疗后复发转移性鼻咽癌60例,分别采用GP方案或FP方案静脉化疗,21天为1周期。结果：GP组：CR5例、PR19例,有效率（CR＋PR）80％;FP组：CR2例、PR14例,有效率（CR＋PR）53．3％两组有效率有显著差异（P=0．0283）。中位PFS（无进展生存期）GP组8个月,FP组3个月（P=0．0001）。中位OS期（总生存期）GP组11个月,FP组7个月（P=0．0002）。两组毒性均能耐受。结论：GP方案较FP可以更好改善复发转移性鼻咽癌的RR、PFS和OS。     

BTD、MPT方案序贯治疗新诊断的非移植多发性骨髓瘤患者的疗效分析

 目的　回顾性总结BTD与MPT方案序贯治疗新诊断的非移植多发性骨髓瘤（MM）患者的临床疗效及不良反应。方法　选取2006年1月至2010年5月应用BTD、MPT方案序贯治疗的新诊断非移植MM患者36例为研究对象,其中BTD方案诱导治疗至少2个疗程,达到部分缓解（PR）及其以上疗效后,改用MPT方案巩固治疗至少2个疗程,再应用小剂量沙利度胺维持治疗,直至疾病复发、进展。观察患者疗效及不良反应。结果　应用BTD方案诱导治疗的36例MM患者中,7例（19.4 %）获得完全缓解（CR）,8例（22.2 %）获得非常好的部分缓解（VGPR）,14例（38.9 %）获得PR,总缓解（OR）率为80.6 %。29例有效（CR+VGPR+PR）患者应用MPT方案维持治疗,1例VGPR患者获得CR,3例PR患者获得VGPR,4例（1例既往CR,1例VGPR,2例PR）出现复发进展,其余患者病情平稳。在应用小剂量沙利度胺维持治疗的25例患者中,3例出现进展,其中2例死亡,其余患者病情稳定。中位随访16.5（2～46）个月,中位无进展生存期（PFS）尚未获得,预期1年生存率为86.0 %,3年生存率为77.0 %。不良反应主要包括血小板减少、周围神经病变、带状疱疹、胃肠道反应、贫血、感染、便秘、疲乏、皮疹等。3～4度不良反应发生率较低。结论　BTD、MPT方案序贯治疗可以用于新诊断的非移植MM患者的一线治疗。     

异基因造血干细胞移植治疗高危多发性骨髓瘤临床分析

目的探讨异基因造血干细胞移植(allo-HSCT)治疗高危多发性骨髓瘤(MM)的效果和预后影响因素。方法回顾性分析2003年4月至2017年3月海军军医大学附属长征医院44例确诊并行allo-HSCT的高危MM患者临床资料。分析患者总有效率、复发率、非复发相关死亡(NRM)率、移植物抗宿主病(GVHD)发生率等;采用Kaplan-Meier法分析患者移植后总生存(OS)、无进展生存(PFS);采用Cox比例风险模型对预后影响因素进行回归分析。结果 44例患者移植后有38例可评估疗效。中位随访时间111个月(0～216个月), 其中22例生存, 22例死亡, 21例复发。移植前完全缓解(CR)率29.5%(13/44), 非常好的部分缓解(VGPR)率45.5%(20/44), 部分缓解(PR)率22.7%(10/44), 疾病稳定(SD)率2.3%(1/44);移植后CR率71.1%(27/38), VGPR率13.2%(5/38), PR率13.2%(5/38), 疾病进展(PD)率2.6%(1/38)。5年OS率、PFS率分别为51.8%、47.8%, 10年OS率、PFS率分别为...     

外周血造血干细胞移植治疗恶性浆细胞病19例临床分析

 目的 评价外周血干细胞移植（PBSCT）治疗恶性浆细胞病的安全性和有效性。方法 1999年3月至2005年8月，用PBSCT治疗恶性浆细胞病19例，其中自体PBSCT（APBSCT）治疗多发性骨髓瘤（MM）16例、华氏巨球蛋白血症（WM）1例，异基因PBSCT（allo-PBSCT）治疗MM 2例。结果 17例APBSCT患者均重建造血，中性粒细胞植入（≥0.5×109／L）和不需血小板输注（≥20×109／L）的时间分别为13（9~24）d和13（8~25）d；移植后完全缓解（CR）或接近完全缓解（nCR）7例（41.2 %），部分缓解（PR）9例（52.9 %），总有效率为94.1 %；平均随访30个月，复发或进展10例，其中死亡7例，中位无进展生存（PFS）期和总生存（OS）期分别为18（2 ~ 69）个月和49（14 ~ 75）个月，3年PFS率和OS率分别为39.4 %和75.5 %。移植相关毒副反应主要为发热9例（52.9 %）、口腔溃疡或糜烂5例（29.4 %）、腹泻4例（23.5 %）、肝功能异常3例（17.6 %）、带状疱疹和肺部巨细胞病毒感染各1例（5.9 %），随着造血重建和对症治疗后均完全恢复。2例allo-PBSCT患者中，1例获CR并已无病生存41个月，1例于移植后7个月死于重度移植物抗宿主病（GVHD）。结论 PBSCT是治疗恶性浆细胞病的有效方法；如何减少APBSCT的复发和提高allo-PBSCT的安全性尚需进一步研究。     

自体造血干细胞移植治疗难治复发霍奇金淋巴瘤、灰区淋巴瘤的疗效及预后分析

 目的　评价自体外周血干细胞移植（APBSCT）对霍奇金淋巴瘤（HL）及灰区淋巴瘤患者的缓解率和生存率的作用。方法　回顾性分析30例接受APBSCT的HL及灰区淋巴瘤患者临床资料,中位移植年龄30岁（13～55岁）,病理类型以结节硬化型HL为主,占19例;临床分期以Ⅲ～Ⅳ期为主;分析APBSCT治疗HL及灰区淋巴瘤患者的疗效及生存情况,并探讨了相关影响因素。结果　30例患者均成功采集干细胞,采集单个核细胞中位数为6.8×108／kg（1.0×108／kg～13.8×108／kg）,CD+34 细胞中位数为6.3×106／kg（0.6×106／kg～20.6×106／kg）。中性粒细胞中位植入时间9 d（8～12 d）。28例可评估患者,中位随访时间为18.5个月（2.5～95.0个月）,18例（64.3 %）获完全缓解（CR）,7例（25.0 %）部分缓解（PR）,总反应率（RR）89.3 %。预计5年总生存（OS）率、无进展生存（PFS）率分别为78 %、58 %。7例未缓解患者在移植前更换化疗方案为利妥昔单抗联合化疗后3例获得CR,2例PR。单因素分析提示移植前疾病状态及更换化疗种类数影响OS,移植前放疗史影响PFS。结论　APBSCT可提高HL及灰区淋巴瘤患者CR率,改善患者的OS及PFS;移植前挽救化疗采用利妥昔单抗联合化疗有助于改善移植前疗效;移植前化疗敏感性影响生存,过多化疗种类更换不利生存,移植前放疗史有影响患者PFS的趋势。     

GP和FP治疗转移性鼻咽癌的疗效观察

目的:观察吉西他滨+顺铂(GP)方案和氟尿嘧啶+顺铂(FP)方案治疗晚期复发转移性鼻咽癌的疗效及不良反应.方法:选择放疗后复发转移性鼻咽癌60例,分别采用GP方案或FP方案静脉化疗,21天为1周期.结果:GP组:CR5例、PR19例,有效率(CR+ PR) 80 %;FP组:CR2例、PR14例,有效率( CR + PR) 53.3%两组有效率有显著差异(P=0.0283).中位PFS(无进展生存期)GP组8个月,FP组 3个月(P=0.0001).中位OS期(总生存期)GP组11个月,FP组7个月(P=0.0002).两组毒性均能耐受.结论:GP方案较FP可以更好改善复发转移性鼻咽癌的RR、PFS和OS.     

造血干细胞移植治疗16例多发性骨髓瘤疗效分析

目的：评价造血干细胞移植（SCT）治疗多发性骨髓瘤（MM）患者的疗效。方法：回顾性分析我院SCT治疗16例D-S分期Ⅲ期MM患者。自体造血干细胞移植（ASCT）13例。2例原发耐药、2例ASCT后复发和1例高危患者接受HLA匹配同胞供者异基因造血干细胞移植（allo-SCT）;其中4例应用清髓性预处理。采用NCI/SWOG（2004年前）和EBMT标准分析治疗反应。结果：共完成ASCT20例次,无移植相关死亡（TRM）。ASCT后均有效,9例获得完全缓解（CR）,4例部分缓解（PR）。10例移植前CR/PR者,中位随访35（6～126）个月,3例复发/进展,中位总生存（OS）尚未达到[已超过35（6～119）个月],中位无进展生存（PFS）27（1～50）个月。3例ASCT前处于疾病稳定状态（SD）者分别于移植后11、7和5个月后复发/进展。Allo-SCT组1例预处理后早期TRM,余3例CR,1例PR。1例患者移植后4个月死于急性移植物抗宿主病（GVHD）合并严重肺感染;余3例生存分别已达39、41和14个月,其中仅后者仍持续PFS。结论：ASCT治疗MM耐受性好,能明显延长PFS和OS;原发难治患者ASCT仍有效,但PFS持续时间短暂。沙利度胺联合治疗用于移植前/后可能提高疗效。allo-SCT有效提高高危及复发/难治患者的治疗反应率。     

PAD序贯自体造血干细胞移植治疗难治多发性骨髓瘤（附22例）

目的：多发性骨髓瘤（multiple myeloma,MM）至今仍不可治愈,几乎所有病人均会出现复发或难治,本文初步探讨硼替佐米应用于难治性MM患者PAD化疗并序贯自体外周血造血干细胞移植（autologous pe-ripheral blood stem cell t ransplantation,APBSCT）的可行性和疗效。方法：采用PAD（硼替佐米＋阿霉素＋地塞米松）方案治疗复发或难治性MM。结果：22例中3例难治MM患者给予PAD方案化疗4-6个疗程后,2例达到接近完全缓解（nCR）,1例达到部分缓解（VGPR）,并随后行APBSCT,动员方案PAD＋CTX（PAD,环磷酰胺1.5g/m2,d15）联合G-CSF。预处理方案为马法兰140mg/m2。移植后采用沙利度胺100mg/天。所有患者在移植前均达到CR或VGPR,干细胞采集充分,安全有效,移植后造血功能均快速顺利重建。无1例死亡。移植后采用沙利度胺维持,随访3-12个月,病情稳定。结论：PAD用于难治MM患者的治疗达CR后,继续序贯进行APBSCT不仅可行,而且PAD不影响正常造血干细胞动员,故采用PAD和序贯用PAD＋CTX动员方案的APBSCT的治疗手段,为难治MM患者的治疗提供新的治疗手段。但对长期生存的改善作用需进一步研究。     

BCD方案不同疗程数对于初治多发性骨髓瘤的疗效影响

  目的  分析2、4、6个疗程以硼替佐米（bortezomib）为基础的3药联合诱导, 包括硼替佐米、环磷酰胺（cyclophosphamide）、地塞米松（dexamethasone）即BCD方案3药联合治疗初治多发性骨髓瘤（multiple myeloma, MM）的疗效。  方法  回顾性分析2014年1月1日至2016年7月30日北京大学人民医院初诊MM一线采用BCD的3药方案治疗≥ 2个疗程、随访资料完整、不合并髓外浆细胞瘤及淀粉样变性的70例患者的临床资料。统计单纯接受2、4、6个疗程患者疾病治疗疗效。  结果  70例患者中, 男女比为36: 34。中位年龄为58.4（33~81）岁, ISS分期Ⅰ、Ⅱ、Ⅱ期分别为16、18、36例。单纯接受2、4、6个疗程BCD患者分别为14、20、36例。仅接受2个疗程组中完全缓解（complete response, CR）、≥非常好的部分缓解（very good partial response, VGPR）、≥部分缓解（partial response, PR）比例分别为14.28%、42.86%和71.42%;仅接受4个疗程组中CR、≥ VGPR、≥ PR比例分别为30.00%、60.00%和80.00%;仅接受6个疗程组中CR、≥ VGPR、≥ PR比例分别为38.89%、75.00%和83.33%。中位随访时间为15.37（2~32）个月, 预估中位无进展生存期（median progression free survival, mPFS）为21.96（95%CI:19.26~24.70）个月。3组无进展生存期（progression free survival, PFS）差异无统计学意义（P=0.700）。  结论  BCD方案3药联合治疗中, 最大疗效发生在4个疗程及以后。随着诱导疗程数的进一步增加, 疾病缓解程度增加, 但总体缓解率提高不明显。一线诱导治疗初诊MM可采用BCD方案4个疗程及以上。      

PAD及VAD样-T方案治疗多发性骨髓瘤126例效果比较

目的：探讨多发性骨髓瘤（MM）行 PAD 方案（硼替佐米、多柔比星、地塞米松）及 VAD 方案（长春新碱、多柔比星／多柔比星衍生物、地塞米松）联合沙利度胺（VAD 样-T 方案）治疗效果的差异。方法回顾性分析54例接受 VAD 样-T 方案及72例接受 PAD 方案治疗的 MM 患者的疗效,包括完全缓解（CR）率、非常好的部分缓解（VGPR）率、总有效率（ORR）、总生存（OS）、无进展生存（PFS）及不良反应发生情况。结果 PAD 组 CR 率高于 VAD 样-T 组,差异有统计学意义[31.5%（23／72）比9.3%（5／54）,χ2＝0.30,P＝0.002],但是 VGPR 率及 ORR 两组相比差异无统计学意义[16.7%（12／72）比16.6%（9／54）,P＝0.180;82.2%（65／72）比81.5%（44／54）,P＝0.190]。 PAD 组中位 PFS 时间长于 VAD 样-T 组[（38.2±2.2）个月比（28.0±7.6）个月,P＝0.017];PAD 组3年 PFS 率和5年 OS 率高于 VAD 样-T 组,但差异均无统计学意义（均 P＞0.05）。 PAD 组末梢神经损害发生率高于 VAD 样-T 组,差异有统计学意义[31.5%（23／72）比14.5%（8／54）,P＝0.03]。结论虽然 VAD 样-T 方案的 CR 率低于 PAD 方案,中位PFS 时间短于 PAD 方案,但 VGPR 率、ORR、3年 PFS 率、5年 OS 率与 PAD 方案相当,且相对安全,末梢神经损害的发生率相对较低。对于国内无法使用硼替佐米及骨髓移植的初发 MM 患者,可选择 VAD 样-T作为一线诱导化疗方案。     

Evaluation of prognostic significance of Revised International Staging System in multiple myeloma北大核心CSCD

Objective: To analyze the influence of Revised International Staging System (RISS) on the prognosis and treatment of multiple myeloma (MM). Methods: The clinical information of two hundred and fifty-nine newly diagnosed patients with MM was retrospectively analyzed. The survival and prognosis of these MM patients was compared by using Durie-Salmon (DS) staging, International Staging System (ISS) and RISS, respectively. The influence of use of bortezomib and autologous stem cell transplantation (ASCT) on the prognosis of patients with MM according to RISS was evaluated. Results: The median age of 259 patients with MM was 58 years (range: 34-87 years). The ratio of male to female was 1.3: 1. The median follow-up time was 32 months (range: 1-188 months), and the median progression-free survival (PFS) and median survival time were 45 and 67 months, respectively. In 239 patients with MM according to DS staging, the patients with DS stage I, II and III MM were accounted for 4.2%, 16.7% and 79.1%, respectively; the median PFS were 68, 41 and 44 months, respectively (P = 0.496), and the median survival time were 99, 64 and 67 months (P = 0.478); the 5-year PFS rates were 60.0%, 38.1% and 31.3% (P = 0.208), and the 5-year overall survival (OS) rates were 60.0%, 60.9% and 53% (P = 0.533). In 236 patients with MM according to ISS, the patients with ISS stage I, II and III MM were accounted for 17.4%, 41.1% and 41.5%, respectively; the median PFS were 53, 48 and 38 months, respectively (P = 0.033), and the median survival time were 68, 92 and 57 months, respectively (P = 0.028); the 5-year PFS rates were 36.8%, 40.0% and 25.6% (P = 0.291), and the OS rates were 60.0%, 63.9% and 42.1%, respectively (P = 0.119). In 173 patients with MM according to RISS, the patients with RISS stage I, II and III MM were accounted for 9.2%, 81.6% and 9.2%, respectively; the median PFS were 68, 47 and 16 months (P = 0.022), and the median survival time were statistics not shown and 72 and 25 months, respectively (P = 0.001). The 5-year PFS rates were 55.6%, 34.7% and 11.1% (P = 0.049), and the OS rates were 80.0%, 59.2% and 22.2% (P = 0.012). The patients with RISS stage III MM had better prognosis in bortezomib-based combination chemotherapy group, and the median survival time was 30 months, which was longer than that of the routine chemotherapy group (14 months, P = 0.014). ASCT had significant difference neither in PFS nor in OS according to RISS (P > 0.05). Conclusion: The RISS is superior to DS staging and ISS in the prognostic significance. Patients with RISS stage III MM have poor prognosis, and the bortezomib can improve the prognosis. Copyright © 2018 by TUMOR. All rights reserved.     

An observational, retrospective analysis of retreatment with bortezomib for multiple myeloma

PurposeThe aim of this retrospective chart review of patients with multiple myeloma (MM) was to describe patterns of retreatment with bortezomib-based therapy and responses to retreatment in a community-based setting.Patients and MethodsData were retrospectively extracted from the medical records of patients treated in US Oncology—affiliated community oncology clinics who received 2 separate treatments with bortezomib-based therapy. Eligible patients had ≥ 60 days between treatments and ≥ 4 bortezomib doses during initial treatment. Responses were determined primarily by laboratory values. Response categories included (1) very good partial response (VGPR), ≥ 90% M-protein decrease; (2) partial response (PR), 50%–89% decrease; and (3) less than PR (< PR), < 50% decrease, excluding progressive disease (PD).ResultsRetreatment response data were available for 82 patients; 5 (6%) had VGPR, 12 (15%) had PR, 52 (63%) had < PR, 5 (6%) had PD, and 8 (10%) died. Among 62 patients with response assessments for initial treatment and retreatment, VGPR/PR rates to retreatment were 44%, 23%, and 13% among patients with VGPR, PR, and < PR to initial treatment, respectively. Median time between bortezomib treatments was 9.7 months; 29% of patients received non-bortezomib therapy between treatments. The most common treatment pattern (58% of patients) was single-agent bortezomib at initial treatment and retreatment. Toxicity contributed to discontinuation in 38% of patients during initial treatment and 22% during retreatment; rates of neuropathy contributing to discontinuation were 18% and 6%, respectively.ConclusionRetreatment with bortezomib-based therapy is feasible, with predictable toxicities. This observational analysis supports bortezomib alone or in combination as a retreatment option after initial bortezomib treatment in patients with relapsed MM.     

Long-Term Results in Multiple Myeloma After High-Dose Melphalan and Autologous Transplantation According to Response Categories in the Era of Old Drugs

BackgroundThe aim of this study was to investigate the correlation between the long-term prognosis of multiple myeloma (MM) and the quality of response to therapy in a cohort of 173 patients treated with high-dose melphalan (HDM) and autologous transplantation in the era of old drugs.Patients and MethodsA total of 173 patients with de novo MM who received a transplant between 1994 and 2010 were analyzed. VAD (vincristine, doxorubicin [Adriamycin], dexamethasone) was used as front-line regimen before auto-HPCT. The conditioning was HDM 200 mg/m². Patients were evaluated for clinical response using the criteria from the European Group for Blood and Marrow Transplantation, modified to include near complete remission (nCR) and very good partial remission (VGPR).ResultsThe response distribution after transplantation in our series was complete remission (CR) in 33 cases (19%), nearly complete remission (nCR) in 38 cases (22%), VGPR in 30 cases (17%), partial remission (PR) in 65 cases (38%), and stable disease (SD) in 7 cases (4%). Patients were followed for 48 ± 36 months. Median overall survival (OS) was not reached for the CR group. Progression-free survival (PFS) was 122 months for CR, 55 months for nCR, 56 months for VGPR, 32 months for PR, and 22 months for SD. Significant differences in PFS and OS were found between the CR and nCR groups (P = .003 and P = .001, respectively), between the CR and VGPR groups (P = .002 and P = .001, respectively), and between the CR and PR groups (P = .000 and P = .001, respectively). Responses were clustered in 3 main categories, ie, CR, nCR + VGPR + PR, and SD. The respective 10-year PFS and OS values were 58% and 70% for CR, 15% and 18% for nCR + VGPR + PR, and 0% and 0% for SD.ConclusionThe achievement of depth and prolonged response represents the most important prognostic factor. The relapse rate is low for patients in CR after 10 years of follow-up, possibly signifying a cure.     

Bortezomib,thalidomide, and dexamethasone as induction therapy for patients with symptomatic multiple myeloma

BACKGROUND:

This single‐center retrospective study determined the efficacy of bortezomib, thalidomide, and dexamethasone (BTD) as induction for patients with multiple myeloma (MM) who were eligible for autologous stem cell transplantation (ASCT).

METHODS:

Patients with symptomatic MM who had received BTD induction before stem cell collection at Winship Cancer Institute were included. BTD induction comprised up to 8 3‐week cycles of bortezomib 1.3 mg/m² on Days 1, 4, 8, and 11; thalidomide 100 mg daily; and dexamethasone 40 mg on Days 1 through 4 and Days 9 through 12. Stem cell mobilization involved granulocyte‐colony–stimulating factor and/or cyclophosphamide. Response was assessed according to European Group for Blood and Marrow Transplantation criteria.

RESULTS:

Review of medical records identified 44 eligible patients (34 patients who were treated in the front‐line setting and 10 patients who were treated for recurrent disease) who received a median of 4 BTD cycles. The overall response rate (ORR) was 91%, which included a greater than or equal to very good partial response (≥VGPR) rate of 57% (including 20% stringent complete responses/complete response [sCR/CR] rate). In front‐line patients, the ORR was 94%, which included a 56% ≥VGPR rate (24% sCR/CR). The median CD34‐positive stem cell collection was 10.67 × 10⁶/kg. The ORR after ASCT in 34 patients who were evaluable for response was 100%, including a 76% ≥VGPR rate (53% sCR/CR). Among all 44 patients, the median progression‐free survival (PFS) was 27.4 months. The median overall survival (OS) was not reached after a median follow‐up of 25 months, and the 2‐year OS rate was 82%. There were no significant differences in PFS (27.4 months vs 23.5 months) or in 2‐year survival (80% vs 90%) between patients who did and did not undergo ASCT, respectively. Twenty patients (45%) developed neuropathy, including 4 (9%) with grade 3 neuropathy episodes, and 1 patient developed deep vein thrombosis.

CONCLUSIONS:

BTD was highly effective and well tolerated as induction for MM patients who were eligible for ASCT. Long‐term outcomes appeared to be similar with or without ASCT consolidation. Cancer 2010. © 2010 American Cancer Society.     

PAD序贯自体造血干细胞移植治疗难治多发性骨髓瘤(附22例)

目的:多发性骨髓瘤(multiple myeloma,MM)至今仍不可治愈,几乎所有病人均会出现复发或难治,本文初步探讨硼替佐米应用于难治性MM患者PAD化疗并序贯自体外周血造血干细胞移植(autologous pe-ripheral blood stem cell t ransplantation,APBSCT)的可行性和疗效。方法:采用PAD(硼替佐米+阿霉素+地塞米松)方案治疗复发或难治性MM。结果:22例中3例难治MM患者给予PAD方案化疗4-6个疗程后,2例达到接近完全缓解(nCR),1例达到部分缓解(VGPR),并随后行APBSCT,动员方案PAD+CTX(PAD,环磷酰胺1.5g/m2,d15)联合G-CSF。预处理方案为马法兰140mg/m2。移植后采用沙利度胺100mg/天。所有患者在移植前均达到CR或VGPR,干细胞采集充分,安全有效,移植后造血功能均快速顺利重建。无1例死亡。移植后采用沙利度胺维持,随访3-12个月,病情稳定。结论:PAD用于难治MM患者的治疗达CR后,继续序贯进行APBSCT不仅可行,而且PAD不影响正常造血干细胞动员,故采用PAD和序贯用PAD+CTX动员方案的APBSCT的治疗手段,为难治MM患者的治疗提供新的治疗手段。但对长期生存的改善作用需进一步研究。     

Long-term outcome in relapsed and refractory multiple myeloma treated with thalidomide. Balancing efficacy and side-effects

A total of 303 MM patients were retrospectively reviewed to evaluate long-term efficacy and toxicity of thalidomide alone or in combination with steroids. Overall response rate was 57% (CR/VGPR 12%). Median TTP, PFS and OS were 13.4 months, 20.6 months, and 26.2 months, respectively. PFS and OS were significantly different according to response (p < 0.0001), with better outcome in patients achieving CR/VGPR (PFS and OS 35.4 months and 63 months, respectively). PFS and OS of patients achieving SD or PR were overlapping (p = 0.3). The addition of steroids significantly increased the response rate (p = 0.01). The most clinically relevant complications were neuropathy (40%), constipation (26%), thromboembolic events (7%). Thalidomide was reduced for toxicity in 68 patients (24%) and permanently discontinued in 36 (12%). In conclusion, thalidomide produces high response rate in relapsed/refractory MM. The best outcome is observed in patients with good quality response, but even patients with suboptimal response may obtain durable survival.     

Real world outcomes with Bortezomib Thalidomide dexamethasone and Cyclophosphamide Bortezomib dexamethasone induction treatment for transplant eligible multiple myeloma patients in a Latin American country. A Retrospective Cohort Study from Grupo Argentino de Mieloma Múltiple

Data about treatment outcomes and toxicity in Latin America are scarce. There are differences with central countries based on access to healthcare system and socioeconomic status. Argentinean Society of Hematology recommends bortezomib-based triplets for induction treatment of transplant eligible newly diagnosed multiple myeloma patients. Most common options are CyBorD (cyclophosphamide, bortezomib and dexamethasone) and VTD (bortezomib, thalidomide and dexamethasone). Main goal of our retrospective, multicentric study was to compare very good partial response rate (VGPR) or better after induction treatment in a real-world setting in Argentina. Secondary objectives included comparison of complete response (CR) post-induction and after bone marrow transplantation, grade 3-4 adverse events (AEs), progression-free survival (PFS) and overall survival (OS). Three hundred twenty-two patients were included (median age at diagnosis: 57 years; 52% male; 28% had ISS3; 14% with high-risk cytogenetics; median follow up: 34 months). CyBorD was indicated in 74% and 26% received VTD. In VTD arm, 72.62% of patients achieved at least VGPR vs 53.36% receiving CyBorD (odds ratio, OR: 1.96 [95% confidence interval, CI: 1.08-3.57; P = .026] after adjusting by age, ISS [International Staging System], lactate dehydrogenase levels (LDH) and cytogenetic risk. Difference in VGPR was 19.26% (95% CI: 15-24). CR rate were 35.92% (VTD) vs 22.55% (CyBorD) (adjusted OR: 2.13 [95% CI: 1.12-4.05]). Difference in CR was 13.37% (95% CI: 9.6-17.53). Adverse events (AEs) were more common with VTD (69.05% vs 55.46% for CyBorD; P = .030), especially grade 3-4 neuropathy (P = .005) and thrombosis (P = .001). Thromboprophylaxis was inadequate in 20.24% of patients. Hematological AEs were more common with CyBorD, especially thrombocytopenia (P = .017). PFS and OS at 24 months were not different between treatments. In this real-world setting, VTD was associated with better CR and VGPR than CyBorD. Nevertheless, CyBorD continues to be the preferred induction regimen in Argentina, based on safety profile. Frontline autologous stem cell transplantation improves quality of responses, especially in countries with limited access to new drugs.