A Systematic Review on Efficacy and Safety of Bortezomib Based Regimens for Treatment of Waldensterom Macroglobulinemia

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 12-12
Author(s):  
Abdul Jabbar Dar ◽  
Qasim Khurshid ◽  
Muhaddis Ejaz Ahmad ◽  
Muhammad Ali Mirza ◽  
Farhan Khalid ◽  
...  
Keyword(s):  
Partial Response ◽  
Response Rate ◽  
Immunoglobulin M ◽  
Cochrane Library ◽  
Published Data ◽  
Efficacy And Safety ◽  
Waldenström Macroglobulinemia ◽  
Waldenstrom Macroglobulinemia ◽  
Major Response ◽  
Number Of Patients

Introduction: Waldenström macroglobulinemia (WM) is a B-cell lymphoproliferative disorder characterized by lymphoplasmacytic bone marrow infiltration and immunoglobulin M (IgM) monoclonal gammopathy. Various studies have shown that the proteasome inhibitor bortezomib (V) targets signaling pathways of relevance in WM. We reported published data on the efficacy profile of (V) for the patients of WM. Methods: We performed a comprehensive literature search on articles following PRISMA guidelines. Beginning with articles published after 2004, we used databases like PubMed, Embase, Clinicaltrials.gov, Cochrane Library and Web of Science. Total 580 articles were identified initially and after detailed screening, we finalized 9 studies involving 375 patients with Waldenstrom Macroglobulinemia (WM) treated by combination of Bortezomib. Results: The total number of patients in the studies involving Bortezomib were 375 and dose of the drug used ranged between 1.3 mg/m2 to 1.6mg/m2. The Complete response (CR) ranged from 0-19% and the partial response (PR) ranged from 44-58%. Bortezomib(V): In the study Treonet et al., N=27, the highest major respose rate was 85% and minimal response rate was 37%. In the study Chen et al., N=27, the major response rate was 26% and the PR was 44%. Progression free survival (PFS) was 16.3 months for Chen et al. Bortezomib(V), Rituximab(R), and Dexamethasone(D): In the study Treon et al., N=23, the highest major response rate was 96%, CR was 13%, Very good partial response (VGPR) was 13%, and PR was 46%. Highest PR and was observed in Dimopolous et al., which was 58% and 42 months respectively. Bortezomib(V), and Rituximab(R): In the study Agathocleus et al., N=42, the highest CR was 14.2-19%. The highest PR was between 47.6-52.3%. In the study Ghobrial et al., N=37, the CR was 3% and PR was 46%. PFS was 15.6 months. Bortezomib(V), Rituximab(R) and Everolimus(E): In the study Ghobrial et al., N=46, the CR was 4%, PR was 46% and PFS was 18 months. Conclusion: Despite heterogenicity in WM patients and various regimens used in literature, Bortezomib containing regimens are a remarkably effective treatment. Combination regimens showed an overall response rate of 96%. The main side effects being anemia, leukopenia, thrombocytopenia, nausea and sensory neuropathies. Although we recommend future randomized prospective trials to better understand the efficacy and safety profile of Bortezomib for WM treatment. Disclosures Anwer: Incyte, Seattle Genetics, Acetylon Pharmaceuticals, AbbVie Pharma, Astellas Pharma, Celegene, Millennium Pharmaceuticals.:Honoraria, Research Funding, Speakers Bureau.

scholarly journals A Systemic Review on Efficacy of Ibrutinib-Based Regimens for the Treatment of Waldenstrom Macroglobulinemia

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 8-9
Author(s):  
Qasim Khurshid ◽  
Abdul Jabbar Dar ◽  
Muhammad Ali Mirza ◽  
Muhaddis Ejaz Ahmad ◽  
Ali Jaan ◽  
...  
Keyword(s):  
Partial Response ◽  
Overall Response Rate ◽  
Response Rate ◽  
Systemic Review ◽  
Cochrane Library ◽  
Progression Rate ◽  
Waldenström Macroglobulinemia ◽  
Overall Response ◽  
Waldenstrom Macroglobulinemia ◽  
Number Of Patients

Introduction: Ibrutinib works by inhibiting downstream signaling after the interaction between the mutated MYD88 (Leu265Pro) protein and BTK. In 2015, Ibrutinib, an inhibitor of Bruton's tyrosine kinase (BTK), was approved by the U.S Food and Drug Administration (FDA) for patients with symptomatic Waldenstrom Macroglobulinemia (WM). Methods: We performed a comprehensive literature search following PRISMA guidelines. Beginning with articles published after April 2015, we used databases like PubMed, Embase, Clinicaltrials.gov, Cochrane Library, and Web of Science. A total of 580 articles were identified initially, and after a detailed screening, we finalized 4 studies involving 248 WM patients. Results: The total number of patients who received Ibratunib based regimens were 248. The dose ranged from 140-560 mg. The overall response rate ranged from 90-92%, very good partial response ranged from 13-23%, and the partial response ranged from 36-75%. 1) Ibtrutunib +Rituximab: In a study by Dimopoulos et al., (N=150), the overall response rate (ORR) was 92% vs 47% in control vs placebo respectively. The progression-free survival (PFS) of 30 months was 82% vs 28% respectively. 2) Ibrutinib alone: In a study by Dimopoulos et al., with (N=31), ORR was 90%, a very good progression rate (VGPR) was 13 %, and the partial response (PR) was 58%. In a study by Treon et al., (N=63) ORR was observed in Treon et al. at 90.50%, VGPR was 15%, PR was 36%, no response (NR) was 57%, with overall survival (OS) was 24 months. In a study by Richard et al., (N=4), PR was noted at 75%. Conclusion: Ibtrutunib used for the treatment of WM showed an overall response rate of 92%. Infections, cytopenia, bleeding, hypertension and atrial fibrillation were major side effects reported. However, due to the recent FDA approval of the drug to be used for the patients of WM there is very limited studies on Ibrutinib. We recommend future randomized prospective trials to understand the efficacy and safety profile of Ibrutinib for WM treatment. Disclosures Anwer: Incyte, Seattle Genetics, Acetylon Pharmaceuticals, AbbVie Pharma, Astellas Pharma, Celegene, Millennium Pharmaceuticals.:Honoraria, Research Funding, Speakers Bureau.

scholarly journals Safety and Efficacy of Purine Analogs for Treatment of Waldenstrom Macroglobulinemia

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 9-10
Author(s):  
Abdul Jabbar Dar ◽  
Qasim Khurshid ◽  
Muhaddis Ejaz Ahmad ◽  
Muhammad Ali Mirza ◽  
Farhan Khalid ◽  
...  
Keyword(s):  
Partial Response ◽  
Progression Free Survival ◽  
Complete Response ◽  
The United States ◽  
Cochrane Library ◽  
Waldenström Macroglobulinemia ◽  
Overall Response ◽  
Waldenstrom Macroglobulinemia ◽  
Purine Analogs ◽  
Number Of Patients

Introduction: There are about 1500 cases every year in the United States of America and while the disease is incurable but it is treatable. Purine analogs are anti-metabolites that mimic the structure of metabolic purines and are used for the treatment of Waldenstrom macroglobulinemia (WM). Methods: We performed a comprehensive literature search on articles following PRISMA guidelines. Beginning with articles published after 1995, we used databases like PubMed, Embase, Clinicaltrials.gov, Cochrane Library and Web of Science. Total 580 articles were identified initially and after detailed screening, we finalized 16 studies involving patients with Waldenstrom macroglobulinemia. Results: The total number of patients in the studies involving regimens based on purine analogs were 1211. The doses of Fludarabine ranged from 25-30 mg/m2 and that of Cladribine ranged from 0.1-0.5 mg/kg. The complete response (CR) observed ranged from 2-15% and the partial response (PR) ranged from 11-67%. The overall response rate (ORR) ranged from 38-95%. Fludarabine: In the study Dhodapkar et al., N=231, the highest CR and ORR was observed, 4% and 66% respectively. In the study Zinzani et al., the PR was 41%. Fludarabine and Cyclophasphamide: In the study Dimopoulos et al., the PR was 55% and the progression free survival (PFS) was 24 months. Fludarabine, Cyclophosphamide and ofatumumab: In the study Gavriatopoulou et al., the PR was 67% and very good partial response (VGPR) was 17% and the PFS was 23 months. Fludarabine, rituximab and cyclophosphamide: In the study Tedeschi et al., the CR was 11.60%, ORR was 79% and the PR was 41.8%. Fludarabine and Rituximab: In the study Treon et al., the CR was 4.6%, VGPR was 32.5%, PR was 48%, and ORR was 95.3% with PFS of 51.2 months. Cladribine: In the study Liu et al., the CR was 5% and PR was 50%. In the studies Dimopoulos et al., 1994 the highest CR was observed, 11.5% with an ORR of 85%. The PR was also the highest, 73%. Conclusion: There is limited literature on regimens containing both Fludarabine and Cladribine, which are used for the treatment of WM. Despite heterogenicity in WM patients and various regimens used in literature. Purine analogs containing regimens are a remarkably effective treatment with overall response rates reaching up to 79%. Neutropenia and thrombocytopenia were the main side effects. There is a paucity of phase 3 randomized trials demonstrating a clinical benefit of anyone regimen over another. We recommend future randomized prospective trials better to understand the efficacy and safety profile of regimens containing purine analogs. Disclosures Anwer: Incyte, Seattle Genetics, Acetylon Pharmaceuticals, AbbVie Pharma, Astellas Pharma, Celegene, Millennium Pharmaceuticals.:Honoraria, Research Funding, Speakers Bureau.

scholarly journals Zanubrutinib for the treatment of MYD88 wild-type Waldenström macroglobulinemia: a substudy of the phase 3 ASPEN trial

2020 ◽  
Vol 4 (23) ◽  
pp. 6009-6018
Author(s):  
Meletios Dimopoulos ◽  
Ramon Garcia Sanz ◽  
Hui-Peng Lee ◽  
Marek Trneny ◽  
Marzia Varettoni ◽  
...  
Keyword(s):  
Partial Response ◽  
Progression Free Survival ◽  
Waldenström Macroglobulinemia ◽  
Activating Mutations ◽  
Waldenstrom Macroglobulinemia ◽  
Mutational Status ◽  
Major Response ◽  
Duration Of Response ◽  
Treatment Naïve ◽  
Poor Outcomes

Abstract Patients with Waldenström macroglobulinemia (WM) lacking activating mutations in the MYD88 gene (MYD88WT) have demonstrated relatively poor outcomes to ibrutinib monotherapy, with no major responses reported in a phase 2 pivotal study. Zanubrutinib is a novel, selective Bruton tyrosine kinase (BTK) inhibitor designed to maximize BTK occupancy and minimize off-target activity. The ASPEN study consisted of a randomized comparison of zanubrutinib and ibrutinib efficacy and safety in patients with WM who have the MYD88 mutation, as well as a separate cohort of patients without MYD88 mutation (MYD88WT) or with unknown mutational status who received zanubrutinib. Results from the latter single-arm cohort are reported herein. Efficacy endpoints included overall, major and complete (CR) or very good partial response (VGPR) rates, progression-free survival (PFS), duration of response (DOR), and overall survival (OS). Twenty-eight patients (23 relapsed/refractory; 5 treatment-naïve) were enrolled, including 26 with centrally confirmed MYD88WT disease and 2 with unknown MYD88 mutational status. At a median follow-up of 17.9 months, 7 of 26 MYD88WT patients (27%) had achieved a VGPR and 50% a major response (partial response or better); there were no CRs. At 18 months, the estimated PFS and OS rates were 68% and 88%, respectively, while the median DOR had not been reached. Two patients discontinued zanubrutinib due to adverse events. Treatment-emergent hypertension, atrial fibrillation, and major hemorrhages were reported in 3, 1 and 2 patients (including 1 concurrent with enoxaparin therapy), respectively. Results of this substudy demonstrate that zanubrutinib monotherapy can induce high quality responses in patients with MYD88WT WM. This trial is registered on www.clinicaltrials.gov as NCT #03053440.

scholarly journals Obinutuzumab and idelalisib in symptomatic patients with relapsed/refractory Waldenström macroglobulinemia

2021 ◽  
Vol 5 (9) ◽  
pp. 2438-2446
Author(s):  
Cécile Tomowiak ◽  
Stéphanie Poulain ◽  
Charles Herbaux ◽  
Aurore Perrot ◽  
Béatrice Mahé ◽  
...  
Keyword(s):  
Partial Response ◽  
Response Rate ◽  
Liver Toxicity ◽  
Univariate Analysis ◽  
Maintenance Phase ◽  
Induction Phase ◽  
Minor Response ◽  
Apparent Lack ◽  
Waldenström Macroglobulinemia ◽  
Waldenstrom Macroglobulinemia

Abstract We present the results of a phase 2 study evaluating the combination of obinutuzumab + idelalisib in relapsed/refractory (R/R) Waldenström macroglobulinemia (WM). The goal was to determine the safety and efficacy of a fixed-duration chemotherapy-free treatment. During the induction phase, patients received idelalisib + obinutuzumab for 6 cycles, followed by a maintenance phase with idelalisib alone for ≤2 years. Forty-eight patients with R/R WM were treated with the induction combination, and 27 patients participated in the maintenance phase. The best responses, reached after a median of 6.5 months (interquartile range, 3.4-7.1; range, 2.6-22.1 months), were very good partial response in 5 patients, partial response in 27 patients, and minor response in 3 patients, leading to overall response rate and major response rate estimates of 71.4% (95% confidence interval [CI], 56.7-83.4) and 65.3% (95% CI, 50.4-78.3), respectively. With a median follow-up of 25.9 months, median progression-free survival was 25.4 months (95% CI, 15.7-29.0). Univariate analysis focusing on molecular screening found no significant impact of CXCR4 genotypes on responses and survivals but a deleterious impact of TP53 mutations on survival. Although there was no grade 5 toxicity, 26 patients were removed from the study because of side effects; the most frequent were neutropenia (9.4%), diarrhea (8.6%), and liver toxicity (9.3%). The combination of idelalisib + obinutuzumab is effective in R/R WM. Nonetheless, the apparent lack of impact of genotype on outcome could give new meaning to targeting of the phosphatidylinositol 3-kinase pathway in WM. This trial was registered at www.clinicaltrials.gov as #NCT02962401.

scholarly journals Long-Term Follow-Up of Ibrutinib Monotherapy in Symptomatic, Previously Treated Patients With Waldenström Macroglobulinemia

2020 ◽  
pp. JCO.20.00555
Author(s):  
Steven P. Treon ◽  
Kirsten Meid ◽  
Joshua Gustine ◽  
Guang Yang ◽  
Lian Xu ◽  
...  
Keyword(s):  
Progression Free Survival ◽  
Response Rates ◽  
Immunoglobulin M ◽  
Waldenström Macroglobulinemia ◽  
Waldenstrom Macroglobulinemia ◽  
Major Response ◽  
Previously Treated Patients ◽  
Previously Treated

PURPOSE We report the long-term findings and final analysis of a pivotal multicenter trial of ibrutinib monotherapy in previously treated patients with Waldenström macroglobulinemia (WM). PATIENTS AND METHODS Sixty-three symptomatic patients with median prior therapies of two (range, one to nine therapies), of whom 40% were refractory to their previous therapy, received ibrutinib at 420 mg/d. Dose reduction was permitted for toxicity. RESULTS The median follow-up was 59 months, and overall and major response rates were 90.5% and 79.4%, respectively. At best response, median serum immunoglobulin M declined from 3,520 to 821 mg/dL, bone marrow disease involvement declined from 60% to 20%, and hemoglobin rose from 10.3 to 14.2 g/dL ( P < .001 for all comparisons). Responses were impacted by mutated (Mut) MYD88 and CXCR4 status. Patients with MYD88Mut, wild-type (WT) CXCR4 showed higher major (97.2% v 68.2%; P < .0001) and very good partial (47.2% v 9.1%; P < .01) response rates and a shorter time to major response (1.8 v 4.7 months; P = .02) versus patients with MYD88Mut CXCR4Mut. Conversely, four patients who had MYD88WT disease showed no major responses. The median 5-year progression-free survival (PFS) rate for all patients was not reached, and was 70% and 38% for those with MYD88Mut CXCR4WT and MYD88Mut CXCR4Mut WM, respectively ( P = .02). In patients with MYD88WT, the median PFS was 0.4 years ( P < .01 for three-way comparisons). The 5-year overall survival rate for all patients was 87%. Grade ≥ 3 adverse events in more than one patient at least possibly related included neutropenia (15.9%), thrombocytopenia (11.1%), and pneumonia (3.2%). Eight patients (12.7%) experienced atrial arrhythmia, and seven of the eight continued therapy with medical management. CONCLUSION Ibrutinib is highly active and produces long-term disease control in previously treated patients with WM. Treatment is tolerable. Response depth, time to major response, and PFS are impacted by MYD88 and CXCR4 mutation status.

scholarly journals Phase II Trial of the Oral Mammalian Target of Rapamycin Inhibitor Everolimus in Relapsed or Refractory Waldenström Macroglobulinemia

2010 ◽  
Vol 28 (8) ◽  
pp. 1408-1414 ◽  
Author(s):  
Irene M. Ghobrial ◽  
Morie Gertz ◽  
Betsy LaPlant ◽  
John Camoriano ◽  
Suzanne Hayman ◽  
...  
Keyword(s):  
Overall Response Rate ◽  
Response Rate ◽  
Single Agent ◽  
Mammalian Target Of Rapamycin ◽  
Signal Transduction Pathway ◽  
Immunoglobulin M ◽  
Target Of Rapamycin ◽  
Waldenström Macroglobulinemia ◽  
Overall Response ◽  
Waldenstrom Macroglobulinemia

Purpose The phosphatidylinositol 3-kinase/mammalian target of rapamycin (mTOR) signal transduction pathway controls cell proliferation and survival. Everolimus is an oral agent targeting raptor mTOR (mTORC1). The trial's goal was to determine the antitumor activity and safety of single-agent everolimus in patients with relapsed/refractory Waldenström macroglobulinemia (WM). Patients and Methods Eligible patients had measurable disease (immunoglobulin M monoclonal protein > 1,000 mg/dL with > 10% marrow involvement or nodal masses > 2 cm), a platelet count more than 75,000 × 106/L, a neutrophil count more than 1,000 × 106/L, and a creatinine and bilirubin less than 2 × the laboratory upper limit of normal. Patients received everolimus 10 mg orally daily and were evaluated monthly. Tumor response was assessed after cycles 2 and 6 and then every three cycles until progression. Results Fifty patients were treated. The median age was 63 years (range, 43 to 85 years). The overall response rate (complete response plus partial remission [PR] plus minimal response [MR]) was 70% (95% CI, 55% to 82%), with a PR of 42% and 28% MR. The median duration of response and median progression-free survival (PFS) have not been reached. The estimated PFS at 6 and 12 months is 75% (95% CI, 64% to 89%) and 62% (95% CI, 48% to 80%), respectively. Grade 3 or higher related toxicities were observed in 56% of patients. The most common were hematologic toxicities with cytopenias. Pulmonary toxicity occurred in 10% of patients. Dose reductions due to toxicity occurred in 52% of patients. Conclusion Everolimus has high single-agent activity with an overall response rate of 70% and manageable toxicity in patients with relapsed WM and offers a potential new therapeutic strategy for this patient group.

scholarly journals P653 Efficacy and safety of an additional 8 weeks of tofacitinib induction therapy: Updated results of the OCTAVE Open study for tofacitinib 8-week induction non-responders

2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S537-S539
Author(s):  
D T Rubin ◽  
M C Dubinsky ◽  
S Danese ◽  
R Saad-Hossne ◽  
D Ponce de Leon ◽  
...  
Keyword(s):  
Clinical Response ◽  
Mucosal Healing ◽  
Published Data ◽  
Efficacy And Safety ◽  
Open Label ◽  
Phase 3 ◽  
Safety Risks ◽  
Number Of Patients ◽  
Factor Inhibitor ◽  
Induction Current

Abstract Background Tofacitinib is an oral, small-molecule JAK inhibitor for the treatment of ulcerative colitis (UC). Efficacy and safety of tofacitinib were demonstrated in three Phase 3, randomised, placebo-controlled trials in patients with moderate to severe UC.1 Patients who received tofacitinib 10 mg twice daily (BID) for 8 weeks (weeks) in OCTAVE Induction 1 and 2 (NCT01465763; NCT01458951) and did not achieve a clinical response (ie induction non-responders [IndNR]) could enter an ongoing, Phase 3, open-label, long-term extension (OLE) study (OCTAVE Open; NCT01470612). We present an update, as of May 2019, of previously published data up to December 2016 from the OLE study for IndNR patients.2 Methods IndNR patients received tofacitinib 10 mg BID in the OLE study. Patients who were still non-responders after an additional 8 weeks of induction were required to discontinue. Clinical response, remission and mucosal healing were evaluated up to Month (M)36 of the OLE study. Adverse events (AEs) and serious AEs (SAEs) are also presented. Results 429 IndNR patients enrolled in the OLE study, 295 of which received tofacitinib 10 mg BID during induction trials (Table). The proportions of patients with prior tumour necrosis factor inhibitor and baseline corticosteroid use were slightly higher in non-responders than responders at M2; other baseline characteristics were generally similar in responders and non-responders. At M2, 59.7%, 25.7% and 16.2% of patients achieved clinical response, mucosal healing and remission, respectively (as observed). Corresponding non-responder imputation and last observation carried forward values were 52.2%, 23.1% and 14.2% (Table). The table shows data up to M36. The proportions of patients with AEs, SAEs and discontinuations due to AEs for IndNR patients censored at M2 (52.2%, 3.7% and 2.4%, respectively) were similar to those for all patients in OCTAVE Induction 1 and 2 (tofacitinib: 55.4%, 3.8% and 3.9%; placebo: 56.4%, 6.0% and 4.3%), with no new safety risks identified (table). Conclusion The majority of patients who did not achieve clinical response to tofacitinib 10 mg BID for 8 weeks in the induction studies – and subsequently received an additional 8 weeks of tofacitinib 10 mg BID in the OLE study—achieved clinical response, with a considerable number of patients in remission and/or mucosal healing at M36. No new safety risks were observed in the additional 8 weeks of induction. These data support extended induction with an additional 8 weeks of tofacitinib for non-responders to 8-week induction. Current product labelling recommends 5 mg BID for maintenance in responders.3,4 References

scholarly journals Comparative efficacy and safety of intra-articular analgesics after knee arthroscopy: a Bayesian network meta-analysis protocol

BMJ Open ◽  
2020 ◽  
Vol 10 (9) ◽  
pp. e035346
Author(s):  
Yuchen He ◽  
Hongyi He ◽  
Dong-Xing Xie ◽  
Xiaoxiao Li ◽  
Yilun Wang
Keyword(s):  
Single Dose ◽  
Pain Relief ◽  
Bayesian Network ◽  
Knee Arthroscopy ◽  
Meta Analysis ◽  
Arthroscopic Surgery ◽  
Cochrane Library ◽  
Efficacy And Safety ◽  
Comparative Efficacy ◽  
Number Of Patients

IntroductionMost of the patients who received arthroscopic knee surgery will suffer moderate to severe pain, which can delay the rehabilitation process and increase the risk of postoperative complications. Therefore, seeking a safe and effective postoperative analgesia is necessary for promoting the application of arthroscopic surgery. This protocol aims to detail a planned systematic review and meta-analysis on the comparative efficacy and safety of single-dose intra-articular injection of analgesics for pain relief after knee arthroscopy.Method and analysisPubMed, Embase, Web of Science and Cochrane Library will be searched from inception to 1 June 2020 to retrieve randomised controlled trials (RCTs) that compared the commonly used single-dose intra-articular analgesics (ie, morphine; bupivacaine (including levobupivacaine); ropivacaine and magnesium alone or in combination) with placebo or between each other for postoperative pain relief among patients who had received knee arthroscopy. The primary outcome is pain intensity at 2-hour and 24-hour postoperatively; the secondary outcomes include side effects (eg, knee effusion, nausea, vomiting and flushing), the number of patients requiring supplementary analgesia and the time to first analgesic request. The methodological quality of the included RCTs will be assessed based on the Cochrane risk of bias table. The Bayesian network meta-analysis will be conducted using WinBUGS V.1.4.3.Ethics and disseminationSince no private or confidential patient data will be contained in the reporting, approval from an ethics committee is not required. Our study raises no ethical issue, and the results will be published in a peer-reviewed journal.PROSPERO registration numberCRD42019130876.

scholarly journals CXCR4 S338X clonality is an important determinant of ibrutinib outcomes in patients with Waldenström macroglobulinemia

2019 ◽  
Vol 3 (19) ◽  
pp. 2800-2803 ◽  
Author(s):  
Joshua N. Gustine ◽  
Lian Xu ◽  
Nicholas Tsakmaklis ◽  
Maria G. Demos ◽  
Amanda Kofides ◽  
...  
Keyword(s):  
Partial Response ◽  
Important Determinant ◽  
Progression Free Survival ◽  
Free Survival ◽  
Waldenström Macroglobulinemia ◽  
Waldenstrom Macroglobulinemia ◽  
Key Points ◽  
Clonality Assessment ◽  
Good Partial Response

Key Points CXCR4 S338X clonality ≥25% is associated with lower very good partial response and shorter progression-free survival to ibrutinib. CXCR4 S338X clonality assessment represents a novel biomarker to predict outcomes to ibrutinib in Waldenström macroglobulinemia patients.

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