The clinical and economic burden of peripheral T-cell lymphoma: a systematic literature review.

Aim: To understand the burden of treatment-naive peripheral T-cell lymphoma (PTCL). Methods: A systematic literature review was conducted in November 2020 following best practice methodology. Results: Fifty-five clinical studies were included, mostly investigating cyclophosphamide, doxorubicin, vincristine and prednisone (CHOP) or 'CHOP-like' regimens, with combination regimens showing similar effectiveness to CHOP alone. Aside from the combination of brentuximab vedotin + cyclophosphamide, doxorubicin and prednisone (A+CHP), other available treatments showed no statistically significant benefit over CHOP in terms of overall or progression-free survival in overall PTCL patients. The mean monthly cost per patient in the USA ranged from 6328 to US$9356 based on six studies. One economic evaluation demonstrated A+CHP to be a more cost-effective treatment option than CHOP. Conclusion: Further research is needed to understand the humanistic and cost impact of frontline treatment for PTCL and its specific subtypes.

Plain language summary Peripheral T-cell lymphoma (PTCL) is an aggressive cancer that develops from white blood cells called T cells, which are an important part of the immune system. There is limited knowledge on the impact PTCL has on patients and their families. This systematic review of 55 clinical studies was conducted to further understand how safe and effective current treatments are for patients with newly diagnosed PTCL, how these treatments and disease impact their quality of life, and the economic impact of treatment and disease. Chemotherapy (cyclophosphamide, doxorubicin, vincristine and prednisone [CHOP]) was the most commonly studied regimen, but had limited effectiveness and a notable side effect profile. A newer treatment option, brentuximab vedotin + cyclophosphamide, doxorubicin and prednisone (A+CHP) was the only treatment to show a significant added benefit over CHOP for patients, with side effects that were comparable to those of CHOP. Six studies assessed the economic impact of PTCL, the majority of which were focused on the USA, and found the mean monthly cost per patient to be 6328­US$9356. No studies were identified that assessed the impact of PTCL or its treatment on quality of life. Further research is needed to understand the impact of frontline PTCL treatment on patients and their families.

Clinical impact of transformation to acute myeloid leukemia in patients with higher-risk myelodysplastic syndromes.

Aim: Forty percent of patients with higher-risk myelodysplastic syndromes (HR-MDS) transform to acute myeloid leukemia (AML). Materials & methods: This retrospective study assessed the impact of HR-MDS transformation to AML on OS in a 6-month landmark analysis and the results were validated using a time-varying analysis. Results: The rate of AML transformation was 26.9% at 1 year. Patients who transformed to AML had a higher risk of death than patients who did not in the 6-month landmark analysis (HR: 1.82; p: 0.0072) and time-varying analysis at 1 year (HR: 2.85; p < 0.0001). Patients treated with azacitidine and decitabine in first-line therapy had similar results. Conclusion: HR-MDS transformation to AML is associated with inferior OS in patients with HR-MDS initiating first-line therapy.

Up to 40% of patients with higher-risk myelodysplastic syndromes (HR-MDS) could experience deterioration or progression to acute myeloid leukemia (AML), a type of blood cancer. We conducted a study to assess whether HR-MDS progression to AML had an unfavorable impact on patient life expectancy after initiating treatment for HR-MDS. Our study concluded that patients who experienced progression to AML had a higher chance of dying immediately after their progression to AML than patients who did not. Patients who received azacitidine and decitabine as their first treatment for HR-MDS had similar results.

Patient preferences for frontline therapies for Philadelphia chromosome-positive acute lymphoblastic leukemia: a discrete choice experiment.

Aim: We examined the preferences of adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) for benefits and risks of tyrosine kinase inhibitors combined with chemotherapy for first-line treatment. Methods: In a discrete choice experiment, 201 patients chose between hypothetical treatment alternatives with varied levels of remission duration and overall survival (OS), and risks of major cardiovascular (CV) events and myelosuppression. Results: Although OS was the most important attribute to patients with Ph+ ALL, they were willing to tolerate a 2.9% increase in CV risk for 1 additional month of OS. Older patients (>59 years) and patients not in remission were less likely to tolerate increased CV risk. Conclusion: Preferences and risk tolerance varied between patients, highlighting the importance of shared decision making when selecting treatments for Ph+ ALL.

Treatments differ in their potential benefits and side effects they may come with. Patients should be involved in deciding which treatments they receive. This is because patients may have different views than physicians on how the benefits and side effects of treatment would affect their quality of life. Additionally, patients may have different risk tolerances. This study shows how patients with a form of leukemia valued survival benefits and side effects of treatments, and the trade-offs that they were willing to make between these. On average, longer survival had most value to patients. They were willing to accept a higher risk of a major cardiovascular side effect (e.g., having a stroke) if the treatment would allow them to live longer. However, not all patients had the same opinion, and some groups of patients were less willing to accept risks to receive longer survival. By involving patients in treatment decisions, we can help ensure they receive treatments that match their personal preferences.

Patient and physician preferences for first-line treatment of classical Hodgkin lymphoma in Germany, France and the United Kingdom.

First-line treatments for classical Hodgkin lymphoma (HL) include ABVD (adriamycin, bleomycin, vinblastine, dacarbazine) and BEACOPPescalated (escalated dose bleomycin, etoposide, adriamycin, cyclophosphamide, vincristine, procarbazine, prednisone). To further improve overall outcomes, positron emission tomography-driven strategies and ABVD or BEACOPP variants incorporating the antibody-drug conjugate brentuximab vedotin (BV) or anti-PD1 antibodies are under investigation in advanced-stage patients. The present study aimed to elicit preferences for attributes associated with ABVD, BEACOPPescalated and BV-AVD (BV, adriamycin, vinblastine and dacarbazine) among patients and physicians. Cross-sectional online discrete choice experiments were administered to HL patients (n = 381) and haematologists/oncologists (n = 357) in France, Germany and the United Kingdom. Included attributes were progression-free survival (PFS), overall survival (OS), and the risk of neuropathy, lung damage, infertility and hospitalisation due to adverse events. Whereas 5-year PFS and OS were the most important treatment attributes to patients, the relative importance of each attribute and preference weights for each level varied among physicians according to the description of the hypothetical patient for whom treatment was recommended. PFS and OS most strongly influenced physicians' recommendations when considering young female patients who did not want children or young male patients. Infertility was more important to physicians' treatment decision than PFS when considering young women with unknown fertility preferences, whereas hospitalisations due to adverse events played the largest role in treatment decisions for older patients.

Achievement of Target A1C <7.0% (<53 mmol/mol) by U.S. Type 2 Diabetes Patients Treated With Basal Insulin in Both Randomized Controlled Trials and Clinical Practice.

OBJECTIVE: Many patients with type 2 diabetes do not reach glycemic goals despite basal insulin treatment. This study assessed the achievement of a target A1C <7.0% (<53 mmol/mol) after initiation of basal insulin in two settings. METHODS: This was a retrospective analysis of pooled randomized controlled trial (RCT) data, from 11 24-week studies of patients initiating basal insulin performed between 2000 and 2005 and of outpatient electronic medical record (EMR) data from the General Electric Centricity database for insulin-naive patients initiating basal insulin between 2005 and 2012. Baseline characteristics stratified by target A1C and fasting plasma glucose (FPG) attainment were compared descriptively. RESULTS: In the RCT dataset, 49.0% of patients failed to achieve the target A1C at 6 months versus 72.4% and 72.9% at 6 and 12 months in the EMR dataset, respectively. Despite this, in the RCT dataset, 79.4% of patients achieved the target A1C and/or an FPG <130 mg/dL. In the EMR dataset, only 47.6% and 47.3% of patients achieved an A1C <7.0% and/or FPG <130 mg/dL at 6 and 12 months, respectively. Overall, patients with an A1C >7.0% had a longer diabetes duration and were more likely to be female, nonwhite, and self-funding or covered by Medicaid. Among patients with an A1C >7.0%, more RCT patients (58.0%) had an FPG <130 mg/dL than EMR patients at 6 months (27.8%) and 12 months (27.7%). CONCLUSION: Unmet needs remain after basal insulin initiation, particularly in real-world settings, where many patients require further insulin titration. In both populations, patients failing to achieve the target A1C despite attaining an FPG <130 mg/dL require interventions to improve postprandial control.

Brentuximab vedotin in patients with relapsed or refractory Hodgkin lymphoma who are Ineligible for autologous stem cell transplant: A Germany and United Kingdom retrospective study.

OBJECTIVE: Brentuximab vedotin (BV) is an anti-CD30 antibody-drug conjugate licensed for the treatment of relapsed/refractory Hodgkin lymphoma (rrHL) following autologous stem cell transplant (ASCT) or at least two prior therapies when ASCT or multiagent chemotherapy is not an option. The objective of this study was to describe real-world outcomes with BV in patients with rrHL considered ASCT ineligible or who refuse ASCT. METHODS: This was a retrospective medical chart review study that enrolled patients ≥18 years old who were initially diagnosed with HL between January 1, 2008 and June 30, 2014, considered ASCT ineligible, and treated in routine care with BV for progressive disease after multidrug chemotherapy regimens. Clinical outcomes included best response to treatment, progression-free survival (PFS), overall survival (OS), and adverse events. RESULTS: A total of 136 patients were included, with a median age of 70 years at initial HL diagnosis. The most common reasons for ASCT ineligibility were comorbidities (74%) and age (57%). Overall response rate was 74%, and PFS and OS were 15.1 and 17.8 months, respectively. Peripheral neuropathy was observed in 9.6% of patients. CONCLUSION: The results of this study provide real-world evidence on the feasibility and effectiveness of BV in elderly or frail ASCT-ineligible patients with rrHL in a real-world setting.

Development and Validation of Algorithms to Identify Statin Intolerance in a US Administrative Database.

OBJECTIVES: To develop and validate algorithms to define statin intolerance (SI) in an administrative database using electronic medical records (EMRs) as the reference comparison. METHODS: One thousand adults with one or more qualifying changes in statin therapy and one or more previous diagnoses of hyperlipidemia, hypercholesterolemia, or mixed dyslipidemia were identified from the Henry Ford Health System administrative database. Data regarding statin utilization, comorbidities, and adverse effects were extracted from the administrative database and corresponding EMR. Patients were stratified by cardiovascular (CV) risk. SI was classified as absolute intolerance or titration intolerance on the basis of changes in statin utilization and/or the occurrence of adverse effects and laboratory testing for creatine kinase. Measures of concordance (Cohen's kappa [κ]) and accuracy (sensitivity, specificity, positive predictive value [PPV], and negative predictive value) were calculated for the administrative database algorithms. RESULTS: Half of the sample population was white, 52.9% were women, mean age was 60.6 years, and 35.7% were at high CV risk. SI was identified in 11.5% and 14.0%, absolute intolerance in 2.2% and 3.1%, and titration intolerance in 9.7% and 11.8% of the patients in the EMR and the administrative database, respectively. The algorithm identifying any SI had substantial concordance (κ = 0.66) and good sensitivity (78.1%), but modest PPV (64.0%). The titration intolerance algorithm performed better (κ = 0.74; sensitivity 85.4%; PPV 70.1%) than the absolute intolerance algorithm (κ = 0.40; sensitivity 50%; PPV 35.5%) and performed best in the high CV-risk group (n = 353), with robust concordance (κ = 0.73) and good sensitivity (80.9%) and PPV (75.3%). CONCLUSIONS: Conservative but comprehensive algorithms are available to identify SI in administrative databases for application in real-world research. These are the first validated algorithms for use in administrative databases available to decision makers.

Adding Rapid-Acting Insulin or GLP-1 Receptor Agonist to Basal Insulin: Outcomes in a Community Setting.

OBJECTIVE: To evaluate real-world outcomes in patients with type 2 diabetes mellitus (T2DM) receiving basal insulin who initiate add-on therapy with a rapid-acting insulin (RAI) or a glucagon-like peptide 1 (GLP-1) receptor agonist. METHODS: Data were extracted retrospectively from a U. S. health claims database. Adults with T2DM on basal insulin who added an RAI (basal + RAI) or GLP-1 receptor agonist (basal + GLP-1) were included. Propensity score matching (with a 1 up to 3 ratio) was used to control for differences in baseline demographics, clinical characteristics, and health resource utilization. Endpoints included prevalence of hypoglycemia, pancreatic events, all-cause and diabetes-related resource utilization, and costs at 1-year follow-up. RESULTS: Overall, 6,718 matched patients were included: 5,013 basal + RAI and 1,705 basal + GLP1. Patients in both groups experienced a similar proportion of any hypoglycemic event (P = .4079). Hypoglycemic events leading to hospitalization were higher in the basal + RAI cohort (2.7% vs. 1.8%; P = .0444). The basal + GLP-1 cohort experienced fewer all-cause (13.55% vs. 18.61%; P<.0001) and diabetes-related hospitalizations (11.79% vs. 15.68%; P<.0001). The basal + GLP-1 cohort had lower total all-cause health care costs ($18,413 vs. $20,821; P = .0002) but similar diabetes-related costs ($9,134 vs. $8,985; P<.0001) compared with the basal + RAI cohort. CONCLUSIONS: Add-on therapy with a GLP-1 receptor agonist in T2DM patients receiving basal insulin was associated with fewer hospitalizations and lower total all-cause costs compared with add-on therapy using an RAI and could be considered as an alternative to an RAI in certain patients with T2DM who do not achieve effective glycemic control with basal insulin.

Real-world evaluation of the effects of counseling and education in diabetes management.

Background. Patient education has long been recognized as a component of effective diabetes management, but the impact of counseling and education (C/E) interventions on health care costs is not fully understood. Objectives. To identify the incidence and type of diabetes C/E received by type 2 diabetes patients and to evaluate associated economic and clinical outcomes. Methods. This retrospective cohort study used the Premier-Optum Continuum of Care database (2005-2009) to compare adult patients with type 2 diabetes receiving C/E to those not receiving C/E (control). The index date was the first C/E date or, in the control cohort, a randomly assigned date on which some care was delivered. Patients had at least 6 months' pre-index and 12 months' post-index continuous health plan coverage. Health care costs and glycemic levels were evaluated over 12 and 6 months, respectively, with adjustment for differences in baseline characteristics using propensity score matching (PSM). Results. Of 26,790 patients identified, 9.3% received at least one C/E intervention (mean age 53 years, 47% men) and 90.7% received no C/E (mean age 57 years, 54% men). Standard diabetes education was the most common form of C/E (73%). After PSM, C/E patients had some improvements in glycemic levels (among those with laboratory values available), without increased risk for hypoglycemia, and incurred $2,335 per-patient less in diabetes-related health care costs, although their total health care costs increased. Conclusions. Despite the low uptake of C/E services, C/E interventions may be associated with economic and clinical benefits at 12 months. Further analyses are needed to evaluate the long-term cost-effectiveness of such initiatives.

Incidence and predictors of venous thromboembolism (VTE) among ambulatory high-risk cancer patients undergoing chemotherapy in the United States.

BACKGROUND: Recent studies suggest that thromboprophylaxis is beneficial in preventing venous thromboembolism (VTE) in cancer outpatients, but this is not widely adopted because of incomplete understanding of the contemporary incidence of VTE and concerns about bleeding. Therefore, the authors examined the incidence and predictors of VTE in ambulatory patients with bladder, colorectal, lung, ovary, pancreas, or gastric cancers. METHODS: Data were extracted from a large health care claims database of commercially insured patients in the United States between 2004 and 2009. Demographic and clinical characteristics of the cancer cohort (N = 17,284) and an age/sex-matched, noncancer control cohort were evaluated. VTE incidence was recorded during a 3-month to 12-month follow-up period after the initiation of chemotherapy. Multivariate analyses were conducted to identify independent predictors of VTE and bleeding. RESULTS: The mean age of the study population was 64 years, and 51% of patients were women. VTE occurred in 12.6% of the cancer cohort (n = 2170) over 12 months after the initiation of chemotherapy versus 1.4% of controls (n = 237; P < .0001); incidence ranged by cancer type from 19.2% (pancreatic cancer) to 8.2% (bladder cancer). Predictors of VTE included type of cancer, comorbidities (Charlson Comorbidity Index score or obesity), and commonly used specific antineoplastic or supportive care agents (cisplatin, bevacizumab, and erythropoietin). CONCLUSIONS: This large, contemporary, real-world analysis confirmed high rates of VTE in select patients with solid tumors and suggested that the incidence of VTE is high in the real-world setting. Awareness of the benefits of targeted thromboprophylaxis may result in a clinically significant reduction in the burden of VTE in this population.

Economic Impact of Transformation to Acute Myeloid Leukemia Among Actively Managed Patients with Higher-Risk Myelodysplastic Syndromes in the United States.

INTRODUCTION: Transformation of higher-risk myelodysplastic syndromes (MDS) to acute myeloid leukemia (AML) may be associated with increased healthcare resource utilization (HCRU) and costs. To describe this economic impact, HCRU and costs were compared between US patients who experienced transformation to AML and those who did not. METHODS: Using the Optum administrative claims data, this retrospective matched cohort study identified patients (≥ 18 years old) with higher-risk MDS who initiated first-line therapy between January 1, 2008, and June 30, 2019. Patients whose disease transformed to AML were matched 1:1 to patients whose disease did not transform, based on the duration of follow-up. The follow-up period was divided into two periods: pre- (before transformation to AML) and post-AML (after transformation to AML). For patients who did not transform to AML, pre- and post-AML periods were determined using the transformation date of their matched pair. HCRU and total adjusted costs (2019 US dollars, $) were compared between patients who transformed to AML and those who did not. RESULTS: A total of 118 matched patient pairs were included in the study. The hospitalization rate was significantly higher in patients who transformed than in those who did not during the entire follow-up (58.8% vs. 44.1%; P = 0.0295) and post-AML (47.5% vs. 28.0%; P = 0.0028) periods. Across all periods, supportive care use was significantly higher among patients who transformed to AML vs. patients who did not transform. Adjusted mean monthly costs for patients with higher-risk MDS who transformed to AML were higher than those who did not transform ($25,964 vs. $19,150; P < 0.0001). The observed total cost difference was more notable in the post-AML period ($36,424 vs. $14,860; P < 0.0001). CONCLUSIONS: Patients with higher-risk MDS whose disease transformed to AML incurred significantly higher healthcare costs compared to those whose disease did not transform, highlighting the important need for treatments that prevent or delay transformation.

A Retrospective Chart Review of Treatment Patterns and Overall Survival among a Cohort of Patients with Relapsed/Refractory Mycosis Fungoides in France, Germany, Italy, Spain and the United Kingdom.

(1) Background: Most patients with mycosis fungoides (MF), a form of cutaneous T-cell lymphoma (CTCL), develop relapsed/refractory (R/R) disease following front-line systemic therapy. This report describes treatment patterns and outcomes from the subpopulation with R/R MF. (2) Methods: This observational, retrospective, cohort study analyzed patient records (1984-2016) from 27 clinical sites in Europe. Outcomes included treatments received, response to first-, second- and third-line treatment, overall survival (OS) and progression-free survival (PFS). (3) Results: Of 104 patients with MF, 100 received second-line and 61 received third-line therapy. The median (range) times from the start of first-line therapy to the first R/R MF and from the first to the second R/R MF were 11.2 (0.3-166.5) and 13.5 (0.0-174.6) months, respectively. Second-and third-line treatment options varied and comprised systemic therapies (85% and 79% of patients, respectively), radiotherapy (32% and 34%, respectively) and topical therapies (48% and 36%, respectively). The median (95% confidence interval [CI]) OS from the diagnosis of the first R/R MF was 11.5 (6.5-not reached [NR]) years and was higher with non-chemotherapy (NR) versus chemotherapy (6.5 years); the estimated median PFS (95% CI) from the time of the first R/R MF was 1.3 (1.0-2.1) years. (4) Conclusions: High rates of R/R disease were observed after second- and third-line treatments in this real-world cohort, with longer median OS in patients receiving non-chemotherapy treatment versus chemotherapy. Following the standard management of MF and using recently approved targeted therapies can help improve patient outcomes in advanced-stage MF.

Impact of venous thromboembolism on mortality of elderly Medicare patients with stage III colon cancer.

BACKGROUND: The improvement in survival rates for patients with colon cancer has shifted the focus from examining cancer-specific mortality to exploring all-cause mortality. Adverse events such as venous thromboembolism (VTE) affect overall survival times and the net clinical benefit of cancer management strategies. METHODS: This retrospective study used Surveillance, Epidemiology and End Results (SEER) Medicare data to examine VTE incidence and mortality rates for elderly patients with stage III colon cancer who were diagnosed in 2004 or 2005 and followed through 2007. The impact of VTE on mortality was estimated using multivariable Cox proportional hazards regression. RESULTS: In all, 20.7% of 4,985 elderly patients with stage III colon cancer had clinically diagnosed VTE following diagnosis. All-cause mortality risk was higher for patients with a VTE diagnosis (hazard ratio [HR]: 1.15, 95% confidence interval [CI]: 1.04-1.27), greater comorbidity burden, more advanced tumor depth and nodal involvement within stage III, advanced age, and male sex; the risk was lower for patients treated with chemotherapy. VTE was associated with higher mortality hazards (HR: 1.41, 95% CI: 1.21-1.64) for patients treated with adjuvant chemotherapy but not for untreated patients. CONCLUSIONS: A new diagnosis of VTE significantly reduced survival rates for elderly patients with stage III colon cancer and further reduced survival rates for patients treated with chemotherapy. Improved prevention and management of VTE for elderly patients with stage III colon cancer who are at risk for VTE is warranted, particularly for patients treated with chemotherapy.

First-line treatment of stage IIB to stage IV classical Hodgkin lymphoma in Italy, Israel, and Spain: Patient characteristics, treatment patterns, and clinical outcomes.

Classical Hodgkin lymphoma (cHL) is curable in 90% of cases, but advanced stage patients who do not respond well to first-line (1L) therapy have poorer outcomes. This retrospective study examines patient characteristics, treatment patterns, clinical outcomes, and safety management of 1L cHL therapies in common clinical practice in Italy (IT), Israel (IL), and Spain (SP). The overall sample (n = 256) included patients with stage IIb to IV cHL, of which 86.3% received ABVD as 1L therapy (n = 221). Clinical outcomes were similar for the overall population and ABVD subsample: complete response (CR) in 75% and 76.5%; 30-month (30-mo) survival (OS) of 92.5% and 93.6%; and 30-mo progression-free survival (PFS) of 70.7% and 72.6%. Thirty-month PFS was significantly lower for patients ≥ 60 years and/or with high (4-7) IPS. Treatment-induced pulmonary and cardiac toxicities, and febrile neutropenia occurred, respectively, in 10%, 2.3%, and 6.8% of ABVD-treated patients. Interim PET or PET-CT scans were performed after two cycles of 1L therapy (PET2) for 70.3% and 66.6% of the overall and ABVD cohorts, respectively. PET2 positive rates were nearly 30% (49/173), yet PET-adapted strategy of dose modification only occurred in a small fraction of patients.

Preferences and Priorities for Relapsed Multiple Myeloma Treatments Among Patients and Caregivers in the United States.

Introduction/Background: This study aimed to describe patient and caregiver preferences for treatments of relapsed or refractory multiple myeloma (MM). Materials and Methods: A survey including discrete-choice experiment (DCE) and best-worst scaling (BWS) exercises was conducted among US patients with relapsed or refractory MM and their caregivers. The DCE included six attributes with varying levels including progression-free survival (PFS), toxicity, and mode and frequency of administration. In addition, the impact of treatment cost was assessed using a fixed-choice question. The BWS exercise included 18 items (modes and frequency of administration, additional treatment convenience, and toxicity items). The survey was administered online to patients recruited from the Multiple Myeloma Research Foundation CoMMpass study (NCT01454297). Results: The final samples consisted of 94 patients and 32 caregivers. Avoiding severe nerve damage was most important to patients, followed by longer PFS. Caregivers considered PFS to be the most important attribute. We estimate that a third or more of patients were cost-sensitive, meaning their treatment preference was altered based on cost implications. Caregivers were not cost-sensitive. The three most bothersome treatment features in the BWS exercise were risk of kidney failure, lowering white blood cell counts, and weakening the immune system. Conclusion: Patients with relapsed or refractory MM and their caregivers consider many factors including efficacy, toxicity, mode/frequency of administration, and cost in their decisions regarding treatment options. The study provides a basis for future Research on patient and caregiver treatment preferences, which could be incorporated into shared decision-making with physicians.

Treatment pathways and clinical outcomes in Hodgkin lymphoma outside Europe and North America: results from the international, multicenter, retrospective, B-HOLISTIC study.

Information on Hodgkin lymphoma (HL) is mostly limited to Europe and North America. This real-world, retrospective study assessed treatment pathways and clinical outcomes in adults with stage IIB-IV classical HL receiving frontline treatment (n = 1598) or relapsed/refractory HL (RRHL, n = 426) in regions outside Europe and North America between January 2010 and December 2013. The primary endpoint was progression-free survival (PFS) in the RRHL group. Among patients with RRHL, 89.0% received salvage chemotherapy; most common regimen was etoposide, methylprednisolone, cytarabine, cisplatin (ESHAP; 26.3%). Median PFS in the RRHL group was 13.2 months (95% confidence interval [CI]: 9.9-20.2) and was longer in patients with vs. without stem cell transplantation (SCT; 20.6 vs. 7.5 months; p = 0.0071). This large-scale study identified a lower PFS for RRHL in the rest of the world compared with Europe and North America, highlighting the need for novel targeted therapies and SCT earlier in the treatment continuum.Clinical trial registration: NCT03327571.

Comparison of incidence/occurrence of cardiovascular events between ponatinib vs bosutinib among patients with at least one prior line of tyrosine kinase inhibitors in chronic myeloid leukemia in a community setting in the United States.

INTRODUCTION: In this real-world study, the incidence of cardiovascular events (CV) including major adverse cardiac events (MACE), arterial occlusive events (AOE), and venous occlusive events (VOE) was evaluated in chronic myeloid leukemia (CML) patients treated with ponatinib or bosutinib in a US commercial database population. MATERIALS AND METHODS: CML patients aged ≥18 years with use of 1 or 2 prior tyrosine kinase inhibitors prescribed bosutinib or ponatinib were selected from the IBM® MarketScan® Research database. Cox proportional hazard model analyses were conducted to examine any difference in CV event risk. RESULTS: Ponatinib and bosutinib was associated with similar incidence and risk of CV events, including MACEs (HR: 1.02; 95% CI: 0.35, 3.01), AOEs (HR: 0.90; 95% CI: 0.43, 1.85) and VOEs (HR: 0.92; 95% CI: 0.44, 1.94). CONCLUSION: Treatment with ponatinib or bosutinib was not associated with significant differences in the incidence of CV events in CML patients.

Contemporary Treatment Patterns and Response in Relapsed/Refractory Cutaneous T-Cell Lymphoma (CTCL) across Five European Countries.

The treatment pattern of cutaneous T-cell lymphoma (CTCL) remains diverse and patient-tailored. The objective of this study was to describe the treatment patterns and outcomes in CTCL patients who were refractory or had relapsed (R/R) after a systemic therapy. A retrospective chart review study was conducted at 27 sites in France, Germany, Italy, Spain and the United Kingdom (UK) of patients who received a first course of systemic therapy and relapsed or were refractory. Data were collected longitudinally from diagnosis to first-, second- and third-line therapy. The study included 157 patients, with a median follow-up of 3.2 years. In total, 151 proceeded to second-line and 90 to third-line therapy. In the first line (n = 147), patients were treated with diverse therapies, including single- and multi-agent chemotherapy in 67 (46%), retinoids in 39 (27%), interferon in 31 (21%), ECP in 4 (3%), corticosteroids in 3 (2%) and new biological agents in 3 (2%). In the second line, the use of chemotherapy and retinoids remained similar to the first line, while the use of new biologics increased slightly. In sharp contrast to the first line, combination chemotherapy was extremely diverse. In the third line, the use of chemotherapy remained high and diverse as in the second line. From the time of first R/R, the median PFS was 1.2 years and the median OS was 11.5 years. The presented real-world data on the current treatments used in the management of R/R CTCL in Europe demonstrate the significant heterogeneity of systemic therapies and combination therapies, as expected from the European guidelines.