新药诱导后自体干细胞移植巩固治疗对不同危险度骨髓瘤患者的作用探讨*

目的:探讨自体造血干细胞移植（autologous hematopoietic stem cell transplantation ,ASCT）作为新药诱导后的巩固治疗对不同危险分层骨髓瘤患者的无进展生存时间（progression-freesurvival ,PFS）及总生存时间（over all survival,OS）的影响。方法:回顾性分析2006年8 月至2011年7 月在本科行自体干细胞移植巩固治疗的67例多发性骨髓瘤患者,根据ISS 分期及FISH检测结果为基础的最新IMWG 预后标准分为高危组17例,中危组24例,低危组26例。另选取同时期67例接受化疗作为巩固治疗的骨髓瘤患者进行年龄、危险分层配对,比较移植组与化疗组的PFS 和OS差异。所有患者前期均接受硼替佐米和/或沙利度胺为主的诱导治疗。结果:所有患者诱导治疗后均达到部分缓解（partial remissive disease ,PR）以上疗效,移植组与化疗组vs . 接近完全缓解率（nCR/CR）差异无统计学意义（44.8% vs. 37.3% ,P=0.380）。 巩固治疗后,高、中、低危移植组患者中位nCR/CR率分别由47.1% ,37.5% ,50.0% 增加为62.9% ,62.5% ,61.5% 。高危患者移植巩固后中位PFS（30.5 个月vs. 11.2 月,P<0.001）和OS（85.5 vs. 34个月,P=0.015）均明显延长;中危移植组和化疗组中位PFS 和OS无统计学差异（P>0.05）;低危移植组患者与化疗组相比,中位PFS 延长（34.8 vs. 17.6 个月,P=0.012）,OS差异无统计学意义（P>0.05）。 结论:在硼替佐米和/或沙利度胺为基础的新药诱导治疗后,高危骨髓瘤患者更能从自体造血干细胞移植巩固治疗中获益,进而延长生存。      

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.     

含利妥昔单抗化疗方案治疗套细胞淋巴瘤效果分析

目的探讨含利妥昔单抗化疗方案治疗套细胞淋巴瘤(MCL)患者效果及预后影响因素。方法回顾性分析2007年6月至2018年11月苏州大学附属第一医院血液科收治的56例≤65岁MCL患者临床资料,化疗方案中均包括利妥昔单抗,观察临床特征、治疗方案及生物学指标对总生存(OS)和无进展生存(PFS)的影响。结果56例患者中位发病年龄57岁,男性43例,女性13例。24例接受R-CHOP方案化疗;29例接受含阿糖胞苷方案化疗,其中15例接受R-hyper CVAD/R-MA方案化疗,14例接受R-CHOP/R-DAHP交替治疗;3例接受其他方案化疗。19例接受自体造血干细胞移植(ASCT)巩固治疗。56例患者中位OS时间74个月,2年OS率83.8%,3年OS率70.9%,2年PFS率72.0%,3年PFS率49.7%。国际预后指数(IPI)评分和治疗中是否接受ASCT是MCL患者OS和PFS的独立影响因素。含阿糖胞苷治疗组总有效率(ORR)93.1%,优于R-CHOP方案组(83.3%),差异无统计学意义(χ²=0.465,P=0.495);两组间OS及PFS差异均无统计学意义(OS:χ²=0.291,P=0.590;PFS:χ²=0.912,P=0.339)。诱导化疗达缓解的MCL患者中,ASCT巩固治疗可延长中位OS时间(72个月比124个月,χ²=3.973,P=0.040)及中位PFS时间(34个月比90个月,χ²=3.984,P=0.046)。简化MCL国际预后指数(sMIPI)评分中高危组患者中接受ASCT巩固治疗患者OS和PFS优于未接受ASCT治疗者(OS:χ²=5.037,P=0.025;PFS:χ²=6.787,P=0.009),而sMIPI评分低危组患者中,是否接受ASCT组间OS、PFS差异均无统计学意义(均P>0.05)。结论含阿糖胞苷的化疗方案对改善MCL患者的预后和生存并不理想。对于诱导化疗达缓解及sMIPI评分中高危组的MCL患者,ASCT巩固治疗可改善其预后,可作为年轻患者的一线巩固治疗方案。     

自体造血干细胞移植治疗多发性骨髓瘤的疗效及预后影响因素分析

目的:探讨自体造血干细胞移植治疗多发性骨髓瘤（MM）的疗效和影响MM患者预后的因素。方法:回顾性分析我院2012年01月至2019年12月37例接受自体造血干细胞移植的MM患者的临床资料,中位随访时间为55（1～91）个月。对37例患者的反应深度、无进展生存时间（PFS）、总生存时间（OS）和影响预后的相关因素进行分析。结果:移植后3月内疗效达到完全缓解率和深度缓解率均优于移植前（P＜0.01,P＜0.05）,移植前后总有效率（ORR）比较无统计学意义（P＞0.05）。患者3年、5年OS率为97.0%、81.4%;3年、5年PFS率为62.7%、51.0%;中位PFS和OS均未获得。单因素分析结果表明移植后3月内疗效获得深度缓解较未获得患者无论是OS 还是PFS均较优 (P均＜0.01);诱导化疗后获得深度缓解可明显延长OS（P＜0.05）;DS分期Ⅰ-Ⅱ期、mSMART3.0危险分层标危患者相比DS分期Ⅲ期、高危患者PFS均有明显优势(P＜0.05)。Cox 多因素回归分析显示,移植后3月内疗效达深度缓解是PFS和OS的独立预后因素,mSMART3.0危险分层也是PFS的独立预后因素。结论:自体造血干细胞移植可以提高 MM 患者的反应深度。移植前后疗效、DS分期和mSMART3.0危险分层均可影响MM患者生存率。移植后3月内疗效达深度缓解是PFS 和OS的独立预后因素,mSMART3.0危险分层也是PFS的独立预后因素。     

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。     

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。     

低剂量硼替佐米联合沙利度胺及化疗治疗多发性骨髓瘤35例

 目的　观察低剂量硼替佐米联合沙利度胺及化疗治疗多发性骨髓瘤（MM）患者的疗效及安全性。方法　35例初治及难治复发MM患者,硼替佐米1.1 mg／m2,第0、3、7、10天,静脉注射;沙利度胺从50 mg／d开始逐渐加量至150 mg／d或患者能够耐受的最大剂量;化疗方案根据每疗程患者情况选择MP、VAD或AD方案。28 d为1个疗程,每例患者至少接受2个疗程以上治疗。达到部分缓解（PR）及以上疗效的患者应用沙利度胺150 mg／d或患者能够耐受的最大剂量维持治疗。采用2006年MM国际统一疗效标准观察疗效,根据国际癌症研究中心不良事件通用命名标准评估不良反应。结果　中位随访20个月,35例患者治疗总有效率82.8 ％,其中完全缓解（CR）率48.6 %,良好的部分缓解（VGPR ）率17.1 %,PR率17.1 %。3年预计无进展生存（PFS）和总生存（OS）率分别为60.92 %和72.41 %。达PR以上疗效患者的OS率高于未达PR患者,差异有统计学意义（P＝0.004）。初治及难治复发患者客观缓解率（ORR）及OS率差异无统计学意义。Ⅲ～Ⅳ度非血液学毒性主要包括乏力（3／35）、恶心、呕吐（8／35）、便秘（4／35）和周围神经病变（3／35）。Ⅲ～Ⅳ度血液学毒性为粒细胞缺乏（10／35）和血小板减少（8／35）。结论　低剂量硼替佐米联合沙利度胺及化疗治疗MM具有较好的疗效及安全性,沙利度胺维持治疗可延长患者PFS时间。     

自体造血干细胞移植治疗多发性骨髓瘤

 自体造血干细胞移植（ASCT）治疗多发性骨髓瘤进展较大。与常规方案相比，ASCT提高了患者的完全缓解（CR）、无事件生存（EFS）和总生存率（OS）。高龄、肾衰竭并非ASCT的禁忌证。双次ASCT可提高CR率，对长期EFS率、OS率影响仍需观察。改进预处理方案、选择移植时机、改进移植后巩固治疗可以提高疗效。     

VD方案联合自体造血干细胞移植治疗难治性不分泌型多发性骨髓瘤一例并文献复习

 目的　观察硼替佐米+地塞米松（VD）方案联合自体造血干细胞移植（ASCT）治疗一例难治性不分泌型多发性骨髓瘤（MM）患者的疗效。方法　通过对1例难治性不分泌型MM患者给予VD方案化疗4个周期,在疾病得到有效控制、临床体征改善后,进行ASCT（预处理方案为美法仑200 mg／m2）,并对国内外相关文献复习。结果　患者经过化疗达到接近完全缓解（nCR）,之后联合ASCT后达到完全缓解（CR）。结论　VD方案联合ASCT可明显改善难治性不分泌型MM患者的临床预后。     

沙利度胺联合MP方案治疗初治多发性骨髓瘤23例

[目的]观察沙利度胺联合MP化疗方案治疗初治多发性骨髓瘤的疗效和不良反应。[方法]2004年2月至2007年1月,47例初治多发性骨髓瘤患者随机分为治疗组（n=23）和对照组（n=24）。两组患者均接受6～8周期的MP方案化疗,治疗组化疗同时接受沙利度胺口服治疗。[结果]治疗组和对照组的总有效率分别是78.3%vs50.0%（P=0.044）。两组2、3年无进展生存率及中位无进展生存时间分别是55.9%vs45.8%、21.0%vs4.2%、27个月vs23个月（P=0.048）。两组2、3年总生存率及中位生存时间分别是86.5%vs83.3%、73.7%vs48.4%、42个月vs33个月（P=0.039）。治疗组血液学毒性和肢体麻木发生率均显著性高于对照组（P〈0.05）。[结论]沙利度胺联合MP方案化疗能提高初治多发性骨髓瘤患者的生存率,延长疾病进展时间;不良反应有所增加,但能耐受。     

30例自体外周造血干细胞移植治疗多发性骨髓瘤的疗效及预后因素评价

目的:对30例多发性骨髓瘤（multiple myeloma,MM）患者经自体外周造血干细胞移植（autologous hematopoietic stem cell transplantation ,APBSCT）治疗后的临床疗效进行评估,并分析可能影响预后的因素。方法:30例MM患者有2 例复发行2 次APBSCT,因此共计移植32例次。移植前予常规联合化疗（11例含万珂）,化疗联合G-CSF 动员APBSC ,选择以马法兰为基础的预处理方案,d0 天回输。结果:动员后患者采集的单个核细胞（MNC）中位数为6.41× 108/kg,CD34+细胞4.75× 106/kg。APBSCT后中位中性粒细胞和血小板重建时间分别为9.5 天和11天。APBSCT后CR和VGPR 率分别为37.5% 和34.4% ,中位生存期（overallsurvival ,OS）为67.27个月,中位无进展生存期（progression-freesurvival ,PFS）为29.77个月,其中CR组、PR组中位PFS 分别为29个月、20个月,VGPR 组中位PFS 未达到,CR+VGPR组与PR组PFS 比较P=0.025。万珂组和非万珂组CR率分别为63.6% 和23.8%（P=0.034）,万珂组中位OS及PFS 均未达到,非万珂组中位PFS 为22个月（P=0.045）。 结论:硼替佐米诱导序贯APBSCT可获得更长的无病生存。APBSCT作为MM诱导缓解后的强化治疗,缓解率高,且移植后获得VGPR 以上反应的患者PFS 获益。      

Beneficial effect of high-dose chemotherapy in multiple myeloma patients with unfavorable prognostic features.

It has been established that high-dose chemotherapy (HDT) improves the therapeutic outcome of patients with multiple myeloma (MM) as compared with standard-dose therapy (SDT); however, little is known about the impact of HDT on different prognostic groups of MM patients. We therefore compared the survival times of 77 patients with previously untreated MM who were enrolled in HDT regimens with those of 64 similar patients <65 years old, who would be eligible for HDT but were treated by SDT. Overall, HDT was superior to SDT with respect to achievement of complete remissions (28% versus 2%; P <0.0001) and improvement of progression-free survival (PFS) (30.2 versus 21.2 months; P = 0.01) as well as overall survival (OS) (median 54.9 versus 49.4 months; P = 0.048). According to the chromosome 13q14 status as determined by fluorescence in situ hybridization and serum levels of beta(2)-microglobulin (beta(2)M), MM patients were separated into a standard-risk group (normal chromosome 13q14 and beta(2)M 4 mg/l). Among patients of the high-risk group, both PFS (26.4 versus 10.7 months; P = 0.004) and OS times (40 versus 23 months; P = 0.05) were longer in patients receiving HDT compared with patients treated by SDT. In the standard-risk group, PFS and OS times were not significantly different between HDT patients and SDT patients. Results of this retrospective analysis suggest that the beneficial effects of HDT are greater in MM patients with high-risk features than in patients with absence of such poor prognostic indicators.     

伴1q21扩增初治多发性骨髓瘤患者疗效及预后的回顾性分析

目的 探讨1q21扩增（1q21+）与初治多发性骨髓瘤（newly diagnosed multiple myeloma,NDMM）患者的治疗疗效及预后的关系,以及1q21+在MM危险分层中的优势。方法 回顾性分析选择2015 年11月至 2021 年11月于首都医科大学附属北京潞河医院就诊的NDMM患者作为研究对象,采用iFISH 技术检测细胞遗传学异常情况,分析1q21+与以硼替佐米为基础的诱导化疗及自体干细胞移植（autologous stem cell transplant,ASCT）疗效及预后的关系,比较MM R2-ISS与R-ISS的分层优势。结果 本研究共纳入159例NDMM患者,1q21+患者79例,非1q21+患者80例,其中41例患者接受RVD方案治疗,118例患者接受非RVD方案治疗。在1q21+的患者中,RVD组的完全缓解（complete remission,CR）率高于非RVD组（P<0.001）;RVD组中,1q21+的CR率低于非1q21+的患者（P=0.004）。多因素分析显示,1q21+是NDMM患者无进展生存期（progression-free survival,PFS）和总生存期（overall survival,OS）的独立危险因素（P<0.05）。在50例接受ASCT的患者中,1q21+患者的1年、3年的OS率及PFS率均较非1q21+患者更低（P=0.055,0.002）。R2-ISS分期Ⅰ期、Ⅱ期、Ⅲ期和Ⅳ期患者的1年PFS率分别为96.3%、95.0%、92.0%和85.4%,3年PFS率分别为88.9%、80.5%、74.0%和61.5%,组间比较差异有统计学意义（P=0.005）。结论 1q21+是NDMM患者OS和PFS的独立危险因素,与患者预后不良有关。RVD方案能提高1q21+ NDMM患者的缓解深度,ASCT可以改善患者预后,但均不能完全克服1q21+带来的不良影响。在临床中R2-ISS分期在MM危险分层方面具有优越性。     

Unsustained complete response of less than 24 months after autologous stem cell transplantation predicts aggressive myeloma with short survival

Complete response (CR) predicts superior survivals in myeloma. To define the impact of duration of CR posttransplantation on survivals, 71 myeloma patients, who underwent an intended early (a staged approach) or frontline use of bortezomib‐based induction, followed by autologous stem cell transplantation (ASCT) were studied. Achievement of CR was assessed every 4‐weekly until maximal response after ASCT and then 6‐weekly thereafter. All patients had follow‐up time of ≥24 months from time of best response, of whom 27 failed to attain CR (non‐CR) whereas 44 achieved CR. At 12, 18 and 24 months post‐ASCT, 3 (4.2%), 6 (8.4%) and 11 (15.4%) patients lost CR, respectively, with maximal survival difference observed in the group with CR durations of ≥24 or <24 months. Patients with unsustained CR had survival inferior to those never achieving CR (p = 0.05). Unsustained CR of <24 months was associated with international staging system stage III (p = 0.007) and shorter postrelapse survival (p < 0.001). Both overall survival and event‐free survival were superior in myeloma patients with CR of ≥24 months (p < 0.001). In multivariate analysis, international staging system stage I/II, CR/nCR post‐ASCT and CR duration of ≥24 months remained favourable prognostic factors for both overall survival and event‐free survival. In conclusion, CR of <24 months is an independent adverse risk factor for survival with a short postrelapse survival. Copyright © 2014 John Wiley & Sons, Ltd.     

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.     

IgD multiple myeloma a descriptive report of 17 cases: survival and response to therapy

Background

Immunoglobulin D multiple myeloma (MM) is rare and has a poorer prognosis than other MM isotypes.

Design and methods

Seventeen patients (pts) diagnosed from 1993 to 2009 with IgD MM were selected from six institutions of Multiple Myeloma Latium-Region GIMEMA Working Group.

Results

Median age was 55 years, 14 patients had bone lesions, eight had renal impairment with estimated glomerular filtration rate (eGFR) < 50 ml/min, one serum calcium ≥ 12 mg/dl, 11 had lambda light chains, five stage III of ISS, six with chromosomal abnormalities. Six pts received conventional chemotherapy (CT): five melphalan + steroids based regimens. Eleven underwent high-doses of chemotherapy with autologous stem cell transplantation (HDT/ASCT), five single and six tandem ASCT: six received bortezomib and/or thalidomide as induction therapy and five VAD. Thalidomide maintenance was used in two pts: one in HDT/ASCT and one in CT group; bortezomib was used in one patient after HDT/ASCT. At a median follow up of 38 (range 19-60) and 50 months (range 17-148) for pts treated with CT and HDT/ASCT, respectively, the overall response rate (ORR) was 83% and 90%. In the group of patients treated with CT, median overall survival (OS) was 34 months (95% CI 15- 54 months), median progression free survival (PFS) was 18 months (95% CI 3-33 months) and median duration of response (DOR) was 7 months (95% CI 5-9 months). Median OS, PFS and DOR were not reached at the time of this analysis in the HDT/ASCT group of patients. Death was observed in 27.3% of pts treated with HDT/ASCT and in 66.7% undergone CT.

Conclusions

Despite the retrospective analysis and the small number of pts our study showed that the use of HDT/ASCT seems to improve also the prognosis of IgD MM patients. Treatment options including new drugs, before and after stem cell transplantation, may further improve the outcomes of these patients.     

Treatment of Multiple Myeloma: A Comprehensive Review

Multiple myeloma (MM) is a neoplastic plasma cell disorder that results in end-organ damage (hypercalcemia, renal insufficiency, anemia, or skeletal lesions). Patients should not be treated unless they have symptomatic (end-organ damage) MM. They should be classified as having high-risk or standard-risk disease. Patients are classified as high risk in the presence of hypodiploidy or deletion of chromosome 13 (del[13]) with conventional cytogenetics, the presence of t(4:14), t(14;16), t(14;20) translocations or del(17p) with fluorescence in situ hybridization. High-risk disease accounts for about 25% of patients with symptomatic MM. If the patient is deemed eligible for an autologous stem cell transplantation (ASCT), 3 or 4 cycles of lenalidomide and low-dose dexamethasone, or bortezomib and dexamethasone, or thalidomide and dexamethasone are reasonable choices. Stem cells should then be collected and one may proceed with an ASCT. If the patient has a complete response or a very good partial response (VGPR), the patient may be followed without maintenance therapy. If the patient has a less than VGPR, a second ASCT is encouraged. If the patient is in the high-risk group, a bortezomib-containing regimen to maximum response followed by 2 additional cycles of therapy is a reasonable approach. Lenalidomide and lowdose dexamethasone is another option for maintenance until progression. If the patient is considered ineligible for an ASCT, then melphalan, prednisone, and thalidomide is suggested for the standard-risk patient, and melphalan, prednisone, and bortezomib (MPV) for the high-risk patient. Treatment of relapsed or refractory MM is covered. The novel therapies—thalidomide, bortezomib, and lenalidomide—have resulted in improved survival rates. The complications of MM are also described. Multiple myeloma is a plasma cell neoplasm that is characterized by a single clone of plasma cells producing a monoclonal protein (M-protein). The malignant proliferation of plasma cells produces skeletal destruction that leads to bone pain and pathologic fractures. The M-protein might lead to renal failure, hyperviscosity syndrome, or through the suppression of uninvolved immunoglobulins, recurrent infections. Anemia and hypercalcemia are common complications.