Jose Ricardo Perez

Persistency with zoledronic acid is associated with clinical benefit in patients with multiple myeloma

Zoledronic acid (ZOL), an intravenous bisphosphonate, has been shown to reduce and delay the incidence of skeletal‐related events (SREs) in multiple myeloma (MM) patients with bone disease. A retrospective claims‐based analysis was conducted that used two distinct US managed care databases to examine the relationship between persistency with ZOL and clinical benefit. Patients >18 years, diagnosed with MM, and with at least one claim for ZOL (or a claim for malignant bone disease and ZOL initiation within 30 days) between 1/1/2001 and 12/31/2006 were included. Patients were evaluated for incidence of SREs and for mortality. Treatment persistency was defined as the absence of a >45 day gap between ZOL administrations. Of 1,655 patients in this analysis, 1,060 received ZOL and 595 received no intravenous bisphosphonate therapy. Compared with patients not receiving bisphosphonate therapy, ZOL‐treated patients had lower incidences of SREs (P < 0.0001) and death (P = 0.0001). Longer persistency with ZOL was associated with lower risks of SREs (P = 0.001), fracture (P = 0.003), and death (P = 0.002) versus shorter persistency. Patients who were persistent with ZOL for ≥1.5 years had an incidence of 15.0 SREs and 6.2 fractures per 100 person‐years. Patients who were persistent for 31–90 days had an incidence of 24.6 SREs and 14.0 fractures per 100 person‐years, and patients not receiving intravenous bisphosphonates had an incidence of 32.2 SREs and 16.9 fractures per 100 person‐years. These data from a real‐world setting indicate that among MM patients, longer persistency with ZOL was associated with a lower risk of SREs and fracture. Am. J. Hematol. 87:490–495, 2012.

Real-World Survival Outcomes and Prognostic Factors Among Patients Receiving First Targeted Therapy for Advanced Renal Cell Carcinoma: A SEER-Medicare Database Analysis

Background The real‐world survival outcomes and prognostic factors among patients receiving first‐line targeted therapy for advanced renal cell carcinoma (aRCC) are not well known. Patients and Methods Adult patients diagnosed with RCC and treated with first‐line targeted therapy were identified from the Surveillance, Epidemiology, and End Results–Medicare database (January 1, 1993 to December 31, 2012). The patients were grouped into early (2006‐2009) or late (2010‐2012) targeted therapy era cohorts by the year of the first‐line targeted therapy initiation. Overall survival (OS) was measured from first‐line targeted therapy initiation and compared between the 2 cohorts using Kaplan‐Meier analyses. The prognostic factors for OS were assessed using a multivariable‐adjusted Cox model. Results A total of 604 and 641 aRCC patients (mean age, 68 years; ˜60% male in both cohorts) initiated first‐line targeted therapy during the early and late targeted therapy eras, respectively. OS was significantly longer in the late than in the early targeted therapy era. Higher tumor grades (hazard ratio [HR], 1.61; 95% confidence interval [CI], 1.31‐2.00) and lung (HR, 1.27; 95% CI, 1.06‐1.53), bone (HR, 1.37; 95% CI, 1.13‐1.66), and liver (HR, 1.42; 95% CI, 1.10‐1.84) metastases were associated with significantly shorter OS. Previous nephrectomy (HR, 0.55; 95% CI, 0.42‐0.72) and pazopanib as first‐line targeted therapy relative to sorafenib (HR, 0.56; 95% CI, 0.37‐0.85) or sunitinib (HR, 0.65; 95% CI, 0.44‐0.95) were associated with significantly longer OS. Conclusion The results of these real‐world analyses suggest progress in aRCC management and identified positive (nephrectomy, pazopanib vs. sunitinib or sorafenib) and negative (higher tumor grade and lung, bone, or liver metastasis) prognostic factors among patients receiving first‐line targeted therapy. Micro‐Abstract Using the Surveillance, Epidemiology, and End Results–Medicare database, we assessed overall survival (OS) among renal cell carcinoma patients who had initiated first‐line targeted therapy. OS was significantly longer in the late (2010‐2012) versus early (2006‐2009) targeted therapy era. Positive (nephrectomy, pazopanib vs. sunitinib or sorafenib) and negative (higher tumor grade and lung, bong, or liver metastases) OS prognostic factors were identified.

Comparative effectiveness of everolimus and axitinib as second targeted therapies for metastatic renal cell carcinoma in the US: a retrospective chart review

Abstract Background Second targeted therapies for metastatic renal cell carcinoma (mRCC) include mammalian target of rapamycin inhibitors (mTORis) and tyrosine kinase inhibitors (TKIs). This observational study compares overall survival (OS) and progression-free survival (PFS) of patients treated with everolimus (an mTORi) and axitinib (a TKI) following first TKI, and assesses the impact of type and duration of first TKI on the relative effectiveness of these second targeted therapies. Methods Retrospective reviews of medical records were conducted by medical oncologists or hematologists/oncologists recruited from a nationwide panel. Included patients with mRCC were required to have discontinued a first TKI (sunitinib, sorafenib, or pazopanib) for medical reasons, and to have initiated everolimus or axitinib as second targeted therapy between February 2012 and January 2013. OS and PFS were compared between patients treated with everolimus vs. axitinib using multivariable Cox proportional hazards regression models. Comparative results were also stratified by type and duration of first TKI. Results Included patients (n = 325 for everolimus and n = 127 for axitinib) had a mean age of 61 years and 31% were female. Sunitinib was the most commonly used first TKI (73%). After adjusting for patient characteristics, no statistically significant differences were observed in OS or PFS between everolimus and axitinib. When stratifying by type and duration of first TKI, there was no statistically significant difference in OS between everolimus and axitinib in all subgroups except for patients with <6 months on sunitinib or sorafenib as first TKI. No significant difference in PFS was observed in any subgroup. Limitations Important limitations include potential missing or inaccurate data in medical charts, and confounding due to unobserved factors. Conclusions In this retrospective chart review, no significant differences were detected in OS or PFS between axitinib and everolimus as second targeted therapy. Longer duration of first TKI was not associated with increased effectiveness of subsequent axitinib compared to everolimus.

Patterns of care among patients receiving sequential targeted therapies for advanced renal cell carcinoma: A retrospective chart review in the USA

To assess real‐world treatment patterns of targeted therapies after failure of first‐line tyrosine kinase inhibitors in patients with advanced renal cell carcinoma.

Real-world dosing and drug costs with everolimus or axitinib as second targeted therapies for advanced renal cell carcinoma: a retrospective chart review in the US

Abstract Objective: To describe dosing patterns and to compare the drug costs per month spent in progression-free survival (PFS) among patients with advanced renal cell carcinoma (aRCC) treated with everolimus or axitinib following a first tyrosine kinase inhibitor (TKI). Methods: A medical record retrospective review was conducted among medical oncologists and hematologists/oncologists in the US. Patient eligibility criteria included: (1) age ≥18 years; (2) discontinuation of first TKI (sunitinib, sorafenib, or pazopanib) for medical reasons; (3) initiation of axitinib or everolimus as a second targeted therapy during February 2012–January 2013. Real-world dosing patterns were summarized. Dose-specific drug costs (as of October 2014) were based on wholesale acquisition costs from RED BOOK Online. PFS was compared between everolimus and axitinib using a multivariable Cox proportion hazards model. Everolimus and axitinib drug costs per month of PFS were compared using multivariable gamma regression models. Results: A total of 325 patients received everolimus and 127 patients received axitinib as second targeted therapy. Higher proportions of patients treated with axitinib vs everolimus started on a higher than label-recommended starting dose (14% vs 2%) or experienced dose escalation (11% vs 1%) on second targeted therapy. The PFS did not differ significantly between patients receiving everolimus or axitinib (adjusted hazard ratio (HR) = 1.16; 95% confidence interval [CI] = 0.73–1.82). After baseline characteristics adjustment, axitinib was associated with 17% (

Results of a Qualitative Study to Develop a Patient Reported Outcome Measure for Patients with 4 Subtypes of Soft Tissue Sarcoma

1830) higher drug costs per month of PFS compared to everolimus (

An Individual Patient Supply Program for Ruxolitinib for the Treatment of Patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (PPV-MF), or Post-Essential Thrombocythemia Myelofibrosis (PET-MF).

12,467 vs

Real-World Effectiveness of Everolimus Subsequent to Different First Targeted Therapies for the Treatment of Metastatic Renal Cell Carcinoma: Synthesis of Retrospective Chart Reviews

10,637; p < 0.001). Limitations: Retrospective observational study design and only drug acquisition costs considered in drug costs estimates. Conclusions: Patients with aRCC receiving axitinib as second targeted therapy were more likely to initiate at a higher than label-recommended dose and were more likely to dose escalate than patients receiving everolimus. With similar observed durations of PFS, drug costs were significantly higher—by 17% per month of PFS—with axitinib than with everolimus.

Comparative effectiveness of everolimus (EVE) and axitinib (AXI) for second-line treatment of metastatic renal cell carcinoma (mRCC) in the United States: A retrospective chart review.

Objective The objective of this research was to develop a disease-specific symptom inventory for soft tissue sarcoma. Methods Literature review and clinical expert and patient interviews were conducted to determine disease-specific symptoms important to patients with one of the four STS subtypes. Clinical experts identified the most relevant STS symptom items from the item pool developed from literature review. Concept elicitation interviews were conducted with patients to elicit their STS symptom experiences followed by a completion of the draft symptom list via web survey. A cognitive interview was conducted on the comprehension and importance of the symptom items. Results Eighty-three symptom items were compiled and discussed with three clinical experts who identified 26 symptoms specific to the four STS subtypes. A total sample of 27 STS participants with self-reported leiomyosarcoma (74%), undifferentiated sarcoma (15%), synovial sarcoma (7%), or liposarcoma (4%) diagnosis completed the web survey and 10 were interviewed. The draft 12-item STS-specific symptom inventory includes abdominal pain, pressure in abdomen, early satiety, bloating, gastrointestinal pain, muscle pain, bone pain, heavy menstrual flow, shortness of breath, chest pain, cough, and painful menstruation. Conclusion A number of symptoms are common across STS subtypes and may form a single STS symptom inventory.