Prognostic value of stringent complete response (sCR) post-autologous stem cell transplant (SCT) in multiple myeloma (MM)

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 8587-8587
Author(s):  
P. Kapoor ◽  
S. Kumar ◽  
A. Dispenzieri ◽  
M. Q. Lacy ◽  
S. R. Hayman ◽  
...  
Keyword(s):  
Plasma Cells ◽  
Response Duration ◽  
Complete Response ◽  
Autologous Stem Cell Transplant ◽  
Cell Transplant ◽  
Entire Cohort ◽  
Best Response ◽  
Prognostic Utility ◽  
Response State ◽  
The Impact

8587 Background: The measurement of serum immunoglobulin free light chains (FLC) has diagnostic and prognostic utility in MM. Normalization of the FLC ratio may define a deeper complete response after therapy than by standard criteria of CR. A sCR requires normalization of the FLC ratio and absence of clonal plasma cells in the bone marrow (BM) in addition to the standard criteria for CR. The objective of our study was to evaluate the impact of specific types of CR [sCR, CR or near CR (nCR/ immunofixation positive CR)] post-SCT on time to progression (TTP) and overall survival (OS). Methods: 468 MM patients who had achieved at least a partial response post-SCT were studied. The results of serum and urine protein studies, serum FLC assay, and BM evaluation, including measurement of marrow plasma cell clonality by immunohistochemistry obtained ≥60 days after SCT were used to determine the best response. TTP was defined as the time from SCT to progression, with non-myeloma related deaths censored. Results: 179 patients achieved varying degrees of CR as their best response. 39, 35 and 105 patients achieved nCR, CR and sCR, respectively. The median estimated follow-up for the entire cohort was 52 months from the diagnosis and 41 months from SCT. The median TTP was 15, 29 and 35 months for patients achieving nCR, CR and sCR, respectively (P<0.0001). The median OS for patients achieving nCR was 53 months from the diagnosis, but not reached for those with a CR or sCR (P=0.0009). The 5-year OS was 80% and 79% for patients with CR and sCR, respectively (P=NS). Similarly, OS from SCT was significantly shorter in patients achieving nCR (42 months vs. not reached for patients in CR and sCR; P<0.001). Conclusions: Achievement of a sCR represents a deeper response state compared to conventional CR, translating to a longer response duration post SCT, validating its inclusion in the modified uniform response criteria. While we did not see a significant improvement in OS with sCR compared to CR in this group, this question needs to be addressed in a larger study. The step wise improvement in the response duration across nCR, CR and sCR highlights the contribution of immunofixation studies, marrow assessment of clonality and FLC estimates. [Table: see text]

scholarly journals Is a severe and early neuropathy related to bortezomib? Is it related to the treatment response? 

2020 ◽  
Vol 8 (2) ◽  
pp. 19-21
Author(s):  
Maria Pereiro Sanchez ◽  
María Perfecta Fernández Gonzalez ◽  
María del Carmen López Doldán ◽  
Aurea María Gómez Marquez ◽  
José Luis Sastre Moral ◽  
...  
Keyword(s):  
Stem Cell ◽  
Standard Treatment ◽  
Stem Cell Transplant ◽  
Plasma Cells ◽  
White Male ◽  
Complete Response ◽  
Autologous Stem Cell Transplant ◽  
Lower Limbs ◽  
Bone Lesions ◽  
Cell Transplant

Myeloma is characterized by the neoplastic proliferation of a clone of plasma cells that invades the bone, causes destruction of the skeleton, and causes bone pain and fractures. In addition, other important features are anemia, hypercalcemia and renal failure. The standard treatment in Spain for autologous stem cell transplant (ASCT)-eligible patients is VTD (bortezomib, thalidomide, dexamethasone). Both bortezomib and thalidomide can cause or exacerbate an existing neuropathy. The mechanism by which they produce it is different in the two drugs.1 A 52-year-old white male was referred to our hospital for the evaluation of anemia (12 g/dL) and serum monoclonal protein (>4 g/dL). The diagnosis was a high cytogenetic risk MM stage R-ISS IIIA, without bone lesions. He only presented mild anemia. He started treatment with standard doses and in accordance to the usual protocol in a candidate patient for autologous stem cell transplant, based on a thalidomide and bortezomib scheme. On the 15th day of the 2nd cycle, the patient showed an autonomic neuropathy and an affectation of the deep sensibility, predominantly the vibratory and proprioceptive with generalized muscle weakness predominant in the lower limbs. He had no pain. He was totally dependent for the basic activities of daily life. Regarding the MM response, the patient showed a strict complete response. This case illustrates a young man with a high cytogenetic risk MM who developed an acute and early polyneuropathy grade IV after 1.5 cycles of standard treatment and with myeloma in strict complete response. The remarkable aspect about this case is the severe and early neuropathy, and an early, deep and persistent myeloma response. On some occasions this relationship has been reported, and we report an example of it.

Risk-adapted maintenance therapy (MaintRx) after ASCT in first-line therapy for multiple myeloma (MM).

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 8607-8607
Author(s):  
Jonathan L. Kaufman ◽  
Joanne Willey ◽  
Michael E. Williams ◽  
Brynn Tiscione ◽  
Arden Buettner ◽  
...  
Keyword(s):  
Induction Therapy ◽  
Plasma Cells ◽  
Autologous Stem Cell Transplant ◽  
Phase Iii ◽  
Cell Transplant ◽  
First Line ◽  
Case Scenario ◽  
First Line Therapy ◽  
Line Therapy ◽  
Best Response

8607 Background: Maintenance lenalidomide improves survival in patients who have undergone induction therapy and then autologous stem cell transplant (ASCT) as first line management for MM. We studied how the extent of cytoreduction after induction therapy à ASCT (as measured by CR, VGPR, or PR as best response) influences MAINTRX prescribing preferences among American Hematology-Oncology physicians (AHOP). Methods: We studied 284 individual AHOPs using a proprietary, live, case-based market research tool to assess prescribing preferences. A core case scenario and variations based on extent of response to induction à ASCT was constructed. Preference data were acquired using blinded audience response technology. All responses for each scenario were obtained contemporaneously prior to any display of respondent selections. All sources of research support were double blinded. The core scenario involved a 59-year-old patient (42% plasma cells in BM, no cytogenetic abnormalities, CrCl 65ml/min, B2M 5.8, PS 1) treated with induction therapy of choice à ASCT. The same query was then posed in each of 3 settings: Following CR (or following VGPR or following PR) as best response, what, if any, further therapy would you recommend now? Results: See Table. Conclusions: Among patients with myeloma getting first-line therapy, MaintRx is almost uniformly preferred by AHOPs. Specific single or doublet therapy preferences are dependent upon best response to induction à ASCT treatment. Compared to the CR setting, preferences for 2 drug MaintRx are significantly increased in patients with VGPR (p <.0.001) or with PR (p < 0.001)] as best overall response. The potential for benefit from intensified MaintRx in these response subsets needs prospective phase III testing. [Table: see text]

Very Good Partial Response and Complete Response Predict Superior Overall Survival and Progression Free Survival After Single Autologous Stem Cell Transplant in Patients with Multiple Myeloma,

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4111-4111
Author(s):  
Victor H Jimenez-Zepeda ◽  
Donna E. Reece ◽  
Suzanne Trudel ◽  
Christine Chen ◽  
Andrew Winter ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Research Funding ◽  
Progression Free Survival ◽  
Complete Response ◽  
Autologous Stem Cell Transplant ◽  
Novel Agents ◽  
Cell Transplant ◽  
Free Survival ◽  
The Impact

Abstract Abstract 4111 In multiple myeloma (MM), the impact of complete response (CR) and very good partial response (VGPR) achievement has been shown mostly after introduction of high dose therapy (HDT) supported by autologous stem cell transplant (ASCT). Recently, the IFM group reported the impact of achievement of CR and VGPR in double ASCT. The purpose of this study is to confirm the prognostic value of CR/VGPR in a large group of patients treated with single ASCT. Methods All consecutive patients who underwent single ASCT at Princess Margaret Hospital between January 2000 and December 2007 were evaluated. Patients were mobilized with cyclophosphamide and G-CSF and majority were conditioned with melphalan 200mg/m2. Response to therapy was assessed according to the IMWC including VGPR. Progression Free Survival (PFS) and Overall Survival (OS) were measured from transplant date to the date of death or last follow-up. OS and DFS were analyzed using the Kaplan-Meier Method. The Cox proportional hazard model was used to assess CR and VGPR and some other prognostic markers at presentation such as age, B2Mg> 460 μmol/L, LDH> 350 IU/L, CRP> 20mg/L, albumin<35g/L and creatinine > 200 μmol/L. All p-values were 2-sided and statistically significant if <0.05. Results 788 patients were identified for the study; their median age was 56 years (30–73). Patient's characteristics are listed in Table 1. Response was assessed at day 100 after ASCT and showed a CR of 6%, PR of 37.5%, and VGPR of 53% (Overall Response rate of 95.5%). Median OS and PFS for the group were 77.43 months and 20.63 months respectively. The median OS and PFS were significantly better for patients who achieved CR/VGPR, 104.5 months versus 51.7 months, and 26.3 months versus 13.53 months respectively. With a median follow-up of 44 months there is no significant difference in OS for those patients who achieved VGPR/CR after induction therapy with novel agents. However, PFS is better in those patients receiving novel agents who achieved VGPR/CR (Median PFS of 24.63months versus 12.4 months respectively (p=0.01). Multivariate analysis shows CR/VGPR as an independent prognostic factor for OS and PFS (Fig 1 and 2). B2Mg> 460 μmol/L, LDH> 350 IU/L, CRP > 20mg/L, albumin<35g/L and creatinine > 200 μmol/L failed to be important factor for survival in the multivariate analysis. Our data suggests that VGPR/CR is clearly important in the pre-novel agents era and for the smaller group of patients who had novel agents induction there is a benefit in PFS and with a longer follow-up perhaps in OS. In conclusion, VGPR/CR remains a simple and powerful indicator in the context of single ASCT and should be considered a relevant objective for MM treatment. Table 1. Clinical characteristics of patients with Multiple Myeloma undergoing single ASCT Clinical characteristic N=788 Median Range % Age (years) 58 31–74 Male 59.4% Female 40.6% Hemoglobin (g/L) 114 54–180 Creatinine (μmol/L) 107 28–1409 B2-microglobulin ((μmol/L) (N=718) 508 260–7270 Albumin (g/L) (N=650) 38 23–54 IgG 51.1% IgA 31.3% IgM 0.4% IgD 0.7% Biclonal 9.9% Not Detected 6.6 Kappa 59.4% Lambda 32.9% Biclonal 2% Not Detected 5.7% Calcium (μmol/L) 2.29 1.62–4.66 LDH (IU/L) (N=754) 235 50–1470 Induction Treatment: 52.2% VAD 22.8% Dexamethasone 6.3% TD 2.3% CP 3.8% DPACE/DTPACE 1.7% DVD 8% CyBORD 2% VD Ab: VAD: Vincristine, Adriamycin and dexamethasone, TD: Thalidomide and Dexamethasone; CP: Cyclophosphamide and Prednisone, DVD: Doxil, Velcade and Dexamethasone, CyBORD: Cyclophosphamide, Bortezomib and Dexamethasone and VD: Valcade and Dexamethasone Disclosures: Jimenez-Zepeda: J & J: Honoraria. Reece:Bristol, Meyers, Squibb: Honoraria, Research Funding; Celgene: Honoraria, Research Funding; Janssen: Honoraria, Research Funding; Johnson&Johnson: Research Funding; Merck: Honoraria, Research Funding; Otsuka: Honoraria, Research Funding; Millennium: Research Funding; Amgen: Honoraria. Chen:Celgene Corporation: Consultancy, Honoraria, Research Funding. Kukreti:Celgene: Honoraria.

Outcomes and toxicities in patients (pts) non-randomly assigned to immunotherapy Children’s Oncology Group (COG) ANBL0032.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 10523-10523
Author(s):  
Ami Vijay Desai ◽  
Andrew Gilman ◽  
Mehmet Fevzi Ozkaynak ◽  
Arlene Naranjo ◽  
Wendy B London ◽  
...  
Keyword(s):  
Partial Response ◽  
Tumor Biology ◽  
Complete Response ◽  
Autologous Stem Cell Transplant ◽  
Cell Transplant ◽  
Entire Cohort ◽  
Gm Csf ◽  
Stage 4 ◽  
Grade 3 ◽  
Clinical Trial Information

10523 Background: Immunotherapy with the anti-GD2 antibody dinutuximab plus sargramostim (GM-CSF), aldesleukin (IL-2) and isotretinoin following consolidation therapy improved outcome for high-risk neuroblastoma (HRNBL) pts enrolled on COG ANBL0032. Randomization was halted in 2009; subsequent pts were non-randomly assigned to immunotherapy. Toxicities and survival were evaluated. Methods: HRNBL pts < 31 years old with a pre-autologous stem cell transplant (ASCT) response of ≥ partial response (PR) were eligible. Demographics, INSS stage, tumor biology, 1993 INRC pre-ASCT response and toxicities were summarized using descriptive statistics. Five-year (yr) EFS and OS from time of study enrollment were estimated. Results: From 2009-2015, 1,183 pts were non-randomly assigned to immunotherapy. 96.7% (n = 1,144) were ≥18 months old and 83.1% (n = 765/921) had stage 4 disease. 45.1% (n = 363/805) of tumors with known biology were MYCN amplified, 94.5% (n = 749/793) had unfavorable histology, and 54.9% (n = 397/723) were diploid. Pre-ASCT, 352 (29.8%) pts had complete response (CR), 418 (35.3%) had very good partial response (VGPR), and 413 (34.9%) had PR. 1,042 (88.1%) pts underwent a single and 141 (11.9%) underwent tandem ASCT. For the entire cohort, 5-yr EFS was 61.1±1.9% and 5-yr OS was 71.9±1.7%. 5-yr EFS and OS for pts ≥18 months of age with stage 4 disease (n = 746) were 58.4±2.3% and 71.0±2.1%. 5-yr EFS and OS were 82.3±4.8% and 86.7±4.2% among pts with stage 3 disease (n = 110). EFS but not OS was superior for those with a CR/VGPR pre-ASCT vs. PR (5-yr EFS: 64.2±2.2% vs. 55.4±3.2%, p = 0.0133; OS: 72.7±2.1% vs. 70.5±2.9%, p = 0.3811). There was a trend toward improved OS for those treated with tandem vs. single transplant (5-yr EFS: 65.9±4.3% vs. 60.4±2.1%, p = 0.1282; OS: 76.5±3.8% vs. 71.2±1.9%, p = 0.0704). Grade ≥3 toxicities ( > 10% of pts) during GM-CSF and IL-2-containing cycles, respectively, included pain (15.6/11.4%), fever (15.1/32.7%), anemia (18.9/21.7%), thrombocytopenia (13.9/17.4%), lymphopenia (12.3/16.0%), and hypokalemia (13.3/25.2%). Additional Grade ≥3 toxicities ( > 10% of pts) included hypoxia (10.1%) during GM-CSF-containing cycles, and anaphylaxis (12.0%), neutropenia (16.1%), hyponatremia (16.5%), and hypotension (13.8%) during IL-2-containing cycles. Conclusions: In this large cohort of HRNBL pts treated with immunotherapy, 5-yr EFS was 61.1%. Superior EFS was observed for pts with stage 3 disease and for those with CR/VGPR pre-ASCT. IL-2-containing cycles were associated with increased toxicity. Clinical trial information: NCT00026312.

Impact of immunoparesis on clinical outcomes following bone marrow transplantation.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e20505-e20505
Author(s):  
Nicholas Torgerson ◽  
Oluwatobi Odetola ◽  
Patrick Hagen ◽  
Nasheed Hossain ◽  
Stephanie Tsai ◽  
...  
Keyword(s):  
Maintenance Therapy ◽  
Conditioning Regimen ◽  
Disease Process ◽  
Progression Free Survival ◽  
Autologous Stem Cell Transplant ◽  
Cell Transplant ◽  
Newly Diagnosed ◽  
Post Transplant ◽  
Best Response ◽  
The Impact

e20505 Background: Immunoparesis (hypogammaglobinemia) has been shown to impact outcomes in patients with newly diagnosed multiple myeloma (MM). Its impact in the post-transplant setting and potential risk of both infections and immune dysregulation impacting relapse is less clear. We sought to assess the role of immunoparesis both pre and post autologous stem cell transplant (ASCT) in newly diagnosed MM patients. Our primary outcome was the impact of immunoparesis on progression free survival (PFS) post-ASCT controlling for maintenance therapy. Methods: We evaluated all MM patients between 2008 and 2017 at Loyola University who underwent ASCT. Inclusion criteria included first ASCT and age > 18 years. We evaluated both the presence of and number of impacted immunoglobulin (Ig) classes pre-transplant and day 100, 6-months, and 12 months post-ASCT. Results: The most common MM isotype was IgG Kappa (41.01%). Immunoparesis pre-ASCT and post-ASCT at day 100, 6 months, and 12 months was seen in 75%, 89.5%, 80%, and 58.5% respectively. Median time from diagnosis to transplant was 7.75 months (5.98 – 13.73). When adjusting for pre-transplant response, conditioning regimen, and post-transplant maintenance, neither the absolute presence of or number of Ig-classes impacting was predictive of hazard of disease progression or death (OS) while ISS-stage, best response post ASCT, and time from diagnosis to transplant were (table). Conclusions: Contrary to studies in the pre-transplant setting, our analysis indicates that immunoparesis is not predictive of relapse or survival in the post-transplant setting, suggesting modification of disease process with treatment and ASCT. When controlled for maintenance therapy, immunoparesis does not increase risk of progression or decrease overall survival alleviating the concern for increased morbidity/mortality secondary to infections in the face of immunoparesis in the era of maintenance therapy. [Table: see text]

Beneficial effect of complete response and type of therapy in multiple myeloma.

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 8541-8541
Author(s):  
Shivlal Pandey ◽  
S. Vincent Rajkumar ◽  
Angela Dispenzieri ◽  
Martha Lacy ◽  
Morie Gertz ◽  
...  
Keyword(s):  
Prognostic Value ◽  
Stem Cell Transplant ◽  
Complete Response ◽  
Autologous Stem Cell Transplant ◽  
Cell Transplant ◽  
Time To Progression ◽  
Entire Cohort ◽  
Disease Biology ◽  
Important Message ◽  
Median Interval

8541 Background: Achievement of a complete response (CR) to treatment is an important predictor of outcome for patients (pts) with myeloma (MM). The goal of the current study was to assess whether the treatment that resulted in CR has any impact on the outcomes. Methods: We identified 462 pts with MM, who fulfilled the IMWG criteria for CR, seen at Mayo Clinic between 1991 and 2011. The treatment was classified into groups by the regimen that led to CR (Table), and also based on whether an autologous stem cell transplant (ASCT) was part of the regimen. The remaining 21 pts had a variety of regimens and are not included in the Table. Results: The median age at diagnosis was 58.5 yrs (27.1–82.3 yrs) with 56% males. The overall survival (OS) from diagnosis for the entire cohort was 10.7 yrs (95% CI; 9.3, NR). The median interval from diagnosis to the recorded CR was 10.3 mos (range 1- 170), with 272 (58.4%) and 385 (82.7%) obtaining a CR in <12 mos and <24 months from diagnosis. We first compared the outcomes based on whether ASCT was part of the regimen; 328 had an ASCT while 117 pts received only chemotherapy. Median time to progression (TTP) following a CR was 5.1 yrs for the ASCT group compared with 5.5 yrs for the rest (P=0.3). Median OS from CR was 9.1 yrs for the ASCT group and 7.5 yrs for the rest (P=0.5) and OS from diagnosis was 10.1 yrs for the ASCT group and 12.6 for the rest (P=0.5). Examining the outcomes based on the regimen utilized showed that the TTP and OS from CR as well as OS from diagnosis were similar for all the groups (Table). Among pts who had a CR within a year of diagnosis, there were no differences in terms of the TTP or OS from the onset of CR or from diagnosis between the ASCT vs. chemotherapy groups or between the different regimens (P=NS for all comparisons). Conclusions: The current study highlights an important message regarding CR in MM. The results suggest that the prognostic value of CR is independent of the nature of therapy, and likely reflects the contribution of disease biology to obtaining a CR. [Table: see text]

Daratumumab (DARA) maintenance or observation (OBS) after treatment with bortezomib, thalidomide and dexamethasone (VTd) with or without DARA and autologous stem cell transplant (ASCT) in patients (pts) with newly diagnosed multiple myeloma (NDMM): CASSIOPEIA Part 2.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 8004-8004
Author(s):  
Philippe Moreau ◽  
Pieter Sonneveld ◽  
Keyword(s):  
Interim Analysis ◽  
Residual Disease ◽  
Progression Free Survival ◽  
Complete Response ◽  
Autologous Stem Cell Transplant ◽  
Second Primary ◽  
Cell Transplant ◽  
Open Label ◽  
Depth Of Response

8004 Background: D-VTd plus ASCT was approved for transplant-eligible (TE) NDMM based on part 1 of CASSIOPEIA. We report a prespecified interim analysis of CASSIOPEIA part 2: DARA maintenance vs OBS in pts with ≥partial response (PR) in part 1, regardless of induction/consolidation (ind/cons) treatment. Methods: CASSIOPEIA is a 2-part, randomized, open-label, phase 3 study in TE NDMM. Pts received 4 cycles ind and 2 cycles cons with D-VTd or VTd. 886 pts who achieved ≥PR were rerandomized to DARA 16 mg/kg IV Q8W for up to 2 yr (n = 442) or OBS (n = 444) until progressive disease per IMWG. Pts were stratified by ind (D-VTd vs VTd) and depth of response (minimum residual disease [MRD] status and post cons response ≥PR). Primary endpoint was progression-free survival (PFS) after second randomization. This interim analysis assessed efficacy and safety after 281 PFS events. A preplanned hierarchical procedure tested key secondary endpoints: time to progression (TTP), ≥complete response (CR), MRD negativity rates by NGS and overall survival (OS). Results: At median follow-up of 35.4 mo, median PFS was not reached (NR) with DARA and 46.7 mo with OBS (HR 0.53; 95% CI 0.42–0.68; P <0.0001). PFS advantage for DARA was consistent across most subgroups. However, a prespecified analysis showed significant interaction with ind/cons treatment arm ( P< 0.0001). PFS HR for DARA vs OBS was 0.32 (95% CI 0.23–0.46) in the VTd arm and 1.02 (0.71–1.47) in the D-VTd arm. Median TTP was NR for DARA vs 46.7 mo for OBS (HR 0.49; 95% CI 0.38–0.62; P <0.0001). More pts in the DARA vs OBS arm achieved ≥CR (72.9% vs 60.8%; OR 2.17; 95% CI 1.54–3.07; P <0.0001). MRD negativity (in ≥CR pts at 10-5) was 58.6% with DARA vs 47.1% with OBS (OR 1.80; 95% CI 1.33–2.43; P= 0.0001). Median OS was NR in either arm. Most common (≥2.5%) grade 3/4 adverse events (AEs) with DARA vs OBS were pneumonia (2.5% vs 1.4%), lymphopenia (3.6% vs 1.8%), and hypertension (3.0% vs 1.6%). Serious AEs occurred in 22.7% (DARA) vs 18.9% (OBS) of pts; the most common (≥2.5%) was pneumonia (2.5% vs 1.6%). 13 (3.0%) pts discontinued DARA due to an AE. The rate of infusion-related reactions was 54.5% (DARA-naïve pts) and 2.2% (prior DARA pts); 90% were grade 1/2.Second primary malignancies occurred in 5.5% (DARA) vs 2.7% (OBS) of pts. Conclusions: CASSIOPEIA part 2 demonstrated a clinical benefit of DARA maintenance in TE NDMM pts, with significantly longer PFS for DARA vs OBS. With current follow-up, maintenance PFS benefit appeared only in pts treated with VTd as ind/cons. Pts who received D-VTd ind/cons with or without DARA maintenance achieved similar PFS; longer follow-up is needed for PFS2 and OS. DARA significantly increased deeper response and MRD negativity rates vs OBS, and was well tolerated with no new safety signals. Clinical trial information: NCT02541383.

scholarly journals Prognostic Significance of Tumor Response at the End of Therapy in Group III Rhabdomyosarcoma: A Report From the Children's Oncology Group

2009 ◽  
Vol 27 (22) ◽  
pp. 3705-3711 ◽  
Author(s):  
David A. Rodeberg ◽  
Julie A. Stoner ◽  
Andrea Hayes-Jordan ◽  
Simon C. Kao ◽  
Suzanne L. Wolden ◽  
...  
Keyword(s):  
Alternative Therapy ◽  
Prognostic Significance ◽  
Complete Response ◽  
Disease Recurrence ◽  
Radiographic Measurement ◽  
Clinical Group ◽  
Group Iii ◽  
Best Response ◽  
Pathology Reports ◽  
The Impact

Purpose Some patients with rhabdomyosarcoma (RMS) achieve less than a complete response (CR) despite receiving all planned therapy. We assessed the impact of best response at the completion of all therapy on patient outcome. Patients and Methods We studied 419 clinical group III participants who completed all protocol therapy without developing progressive disease for Intergroup Rhabdomyosarcoma Study (IRS) IV. Response (complete resolution [CR], partial response [PR; ≥ 50% decrease], or no response [NR; < 50% decrease and < 25% increase]) was determined by radiographic measurement and categorized by the best response. Results At the end of therapy, 341 participants (81%) achieved a best response of CR and 78 (19%) had a best response of PR/NR. Five-year failure-free survival was similar for participants achieving CR (80%) and PR/NR (78%). After adjustment for age, nodal status, primary site, and histology, there was no significant indication of lower risk of failure (hazard ratio [HR], 0.77; 95% CI, 0.46 to 1.27; P = .3) nor death (HR, 0.63; 95% CI, 0.36 to 1.09; P = .1) for CR versus PR/NR participants. Seventeen participants with a best response of PR/NR had surgical procedures; eight (50%) of 16 with available pathology reports had residual viable tumor and only three achieved a complete resection. Resection of residual masses was not associated with improved outcome. Conclusion CR status at the end of protocol therapy in clinical group III participants was not associated with a reduction of disease recurrence and death. Aggressive alternative therapy may not be warranted for RMS patients with a residual mass at the end of planned therapy.

scholarly journals Sequential response-driven bortezomib-based therapy followed by autologous stem cell transplant in AL amyloidosis

2020 ◽  
Vol 4 (17) ◽  
pp. 4175-4179
Author(s):  
Marco Basset ◽  
Paolo Milani ◽  
Mario Nuvolone ◽  
Francesca Benigna ◽  
Lara Rodigari ◽  
...  
Keyword(s):  
Stem Cell ◽  
Partial Response ◽  
Stem Cell Transplant ◽  
Complete Response ◽  
Autologous Stem Cell Transplant ◽  
Survival Duration ◽  
Al Amyloidosis ◽  
Cell Transplant ◽  
Duration Of Response ◽  
Landmark Analyses

Abstract Autologous stem cell transplant (ASCT) is highly effective in selected patients with light chain (AL) amyloidosis. Bortezomib, preceding or following ASCT, improves responses. Satisfactory responses, including at least a partial response, very good partial response (VGPR) with organ response, or complete response, can be observed after induction therapy alone. We report 139 patients treated upfront with cyclophosphamide/bortezomib/dexamethasone (CyBorD), followed by ASCT only if response was unsatisfactory. Only 1 treatment-related death was observed. After CyBorD, hematologic response (HR) rate was 68% (VGPR or better, 51%), with 45% satisfactory responses. Transplant was performed in 55 (40%) subjects and resulted in an 80% HR rate (65% ≥ VGPR). Five-year survival was 86% and 84% in patients treated with ASCT or CyBorD alone, respectively (P = .438). Also, 6- and 12- month landmark analyses did not show differences in survival. Duration of response was not different in the 2 groups (60 vs 49 months; P = .670). Twenty-one (15%) patients with an unsatisfactory response to CyBorD could not undergo ASCT because of ineligibility or refusal; instead, they received rescue chemotherapy, with HR in 38% of cases and 51% 5-year survival. This sequential response-driven approach, offering ASCT to patients who do not attain satisfactory response to upfront CyBorD, is very safe and effective in AL amyloidosis.

Resource Utilization, Costs and Type of Malignancy Associated with Prolonged Length of Stay in Autologous Stem Cell Recipients.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 5311-5311
Author(s):  
Linda J. Patchett ◽  
John M. Hill ◽  
Thomas F. Fitzmaurice ◽  
Kenneth R. Meehan
Keyword(s):  
Stem Cell ◽  
Length Of Stay ◽  
Resource Utilization ◽  
Infection Rate ◽  
Stem Cell Transplant ◽  
Autologous Stem Cell Transplant ◽  
Cell Transplant ◽  
Prolonged Length ◽  
Hospitalization Costs ◽  
The Impact

Abstract In order to contain costs, MDs must first identify the clinical factors contributing to increased resource utilization associated with an autologous stem cell transplant. We performed a retrospective clinical and cost analysis of all autologous transplants performed at Dartmouth- Hitchcock Medical Center over a 30 month period (2002-2004) and identified patients who had a prolonged length of stay &gt; 25 d (PLOS). We pinpointed the clinical characteristics and hospital course of each patient to identify trends. The hospital cost-accounting system highlighted resource utilization and costs of the transplants, allowing a comparison between patients with a PLOS and all other transplant patients. PROLONGED LENGTH OF STAY (PLOS) Results: All Patients LOS &lt; 25 days LOS &gt; 25 days # of patients 87(100%) 58 (67%) 29 (33%) LOS (days) Mean (Median) 24 (22) 20 (20) 31 (31) DISEASE (n = no. of patients) AML 14 9 5 HD/NHL 44 24 20 MM 28 25 3 Other (ITP) 1 1 ENGRAFTMENT (median) ANC &gt; 500 (Platelets &gt; 20K) 12 (18) 11 (16) 13 (27) TRANSFUSIONS UNITS /PT (median) RBC /Platelets &gt; 20 4 (3) 3 (2) 7 (7) PARENTAL NUTRITION (TPN) # of days (median) 9 6 14 TOXICITIES &gt;= GRADE 3 NCI (Common Toxicitity Criteria) Nausea and Vomiting 36% 77% Diarrhea 9% 45% Mucositis 36% 41% Anorexia 57% 83% INFECTION RATE 10% 34% ICU TRANSFER 3% 3% Major contributors to costs included nursing/daily room charge costs (39%), pharmacy (39%), Blood Bank (6%), Laboratory (12%), and other costs (3%). The average daily costs are $4252. The PLOS cohort had grade &gt; 3 toxicity, increased infection rate, engrafted later and required more transfusional support. 1 pt was transferred to the ICU for temporary management. Of the 29 patients identified with PLOS, none died and all were discharged from the hospital. 45% of NHL/HD patients experienced a prolonged LOS, representing 68% of the PLOS cohort. The median LOS&lt;25d is 20d and the median LOS&gt;25d is 30.5d. At an average daily cost of $4252, these additional 10.5 days of hospitalization costs are substantial. Based on these findings, identification of factors underlying PLOS in the NHL/HD cohort may provide the key to minimizing cost of autologous stem cell transplant. Accordingly, we are assessing the impact of age, number of pre-transplant treatment regimens, number of peripheral blood stem cells reinfused, use of IL-2 for post-transplant immune modulation, and the day 15 absolute lymphocyte count on LOS in this population.

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