Time burden and logistical intensity of early-phase clinical trials (EP-CTs).

2021 ◽  
Vol 39 (28_suppl) ◽  
pp. 84-84
Author(s):  
Sienna Durbin ◽  
Debra Lundquist ◽  
Rachel Jimenez ◽  
Megan Healy ◽  
Andrew Johnson ◽  
...  
Keyword(s):  
Metastatic Disease ◽  
Massachusetts General Hospital ◽  
Patient Characteristics ◽  
Trial Participation ◽  
Antibody Drug Conjugate ◽  
Trial Site ◽  
Investigational Agent ◽  
Visit Frequency ◽  
Time Burden ◽  
Early Phase Clinical Trials

84 Background: EP-CTs are increasingly important options for patients with cancer and often involve intensive monitoring. Thus, characterizing the time burden and logistical intensity of EP-CTs could help patients and clinicians make informed decisions regarding trial participation. Methods: We retrospectively reviewed the electronic health records of consecutive patients enrolled in EP-CTs at Massachusetts General Hospital from 2017-2019 to obtain baseline characteristics (demographics and clinical factors), EP-CT investigational agent (immunomodulatory therapy [IM], targeted inhibitor(s) [TI], antibody drug conjugate [ADC]/chemotherapy prodrug), and logistical intensity (trial visit frequency, presence of extended visits, distance traveled in one direction from home zip code to trial site). We defined visit frequency as the number of visits per protocol within the first 28 days on trial. We defined an extended visit as six or more hours in clinic on at least one day during the first 28 days on study. We investigated associations among patient characteristics, investigational agent, and logistical intensity. Results: Among 421 patients (median age=60.6 years, 55.8% female, 97.4% metastatic disease), most (73.6%) had two or more sites of metastatic disease. EP-CTs included 43.2% IM, 43.0% TI, and 13.8% ADC/chemotherapy prodrug. Patients enrolled in ADC/prodrug trials had the highest burden of metastatic disease (mean sites: 2.8 [ADC] vs 2.4 [TI] vs 2.3 [IM], p = 0.007) and oldest age (mean years: 64.0 [ADC] vs 61.7 [IM] vs 58.5 [TI], p = 0.003). Patients enrolled on TI trials had the highest visit frequency compared with those enrolled on other trials (mean visits: 5.5 [TI] vs 5.3 [ADC] vs 5.0 [IM], p = 0.027) and the fewest days spent on trial (mean days: 78.3 [TI] vs 102.2 [IM] vs 131.8 [ADC], p = 0.003). Patients enrolled on TI trials were also most likely to have an extended visit (82.3% [TI] vs 58.2% [IM] vs 29.3% [ADC], p < 0.001) and least likely to receive first in human therapy (38.1% [TI] vs 74.1% [ADC] vs 74.2% [IM], p < 0.001). Distance traveled from home to clinic did not significantly differ across trial type (median miles traveled: 35.1 [TI] vs 34.1 [IM] vs 33.2 [ADC], p = 0.884). Conclusions: In this cohort of patients participating in EP-CTs, we found that a plurality enrolled in IM studies. Those receiving ADC/prodrug regimens were older and had a higher burden of disease. On average, patients participating in EP-CTs had over five visits in the first month, with those enrolled on TI trials having the highest visit frequency and greatest likelihood of extended visits. Patients on TI trials also spent the fewest total days on trial. Despite the lack of significant differences in distance traveled, most patients were still traveling over 30 miles to get to the trial site. These data highlight the time burden and logistical intensity of various EP-CTs, which may help inform patient-clinician discussions about trial participation.

Hospitalizations and emergency department use in cancer clinical trial patients.

2015 ◽  
Vol 33 (29_suppl) ◽  
pp. 160-160
Author(s):  
Ryan David Nipp ◽  
Elizabeth Powell ◽  
Beverly Moy
Keyword(s):  
Health Care ◽  
Emergency Department ◽  
Health Care Utilization ◽  
Metastatic Disease ◽  
Massachusetts General Hospital ◽  
Patient Characteristics ◽  
Care Utilization ◽  
Care Needs ◽  
Hematologic Cancer ◽  
Ed Visits

160 Background: Cancer clinical trials (CTs) often represent the best available treatment for many patients, but little is known about the health care utilization of these patients. We examined correlates of hospitalizations and emergency department (ED) use in cancer CT patients to determine those at greatest risk for these outcomes. Methods: We prospectively collected data on patient characteristics, hospitalizations and ED use among all patients enrolled in cancer CTs at Massachusetts General Hospital in 2014. We calculated the number of hospitalizations and ED visits in the 6-month interval following patients’ CT enrollment. We used linear regression with purposeful selection of covariates to identify factors associated with hospitalizations and ED use. Results: Of 1,218 CT patients (mean age = 58 years; 575 (47%) male), 781 (64%) were married and 851 (70%) had metastatic disease. All cancer types were represented, but hematologic cancers (21%) were most common. Within 6 months following CT enrollment, 519 (43%) and 327 (27%) had at least one hospitalization and ED visit, respectively. At any time during their cancer course, 177 (15%) received a palliative care (PC) consult. Controlling for presence of metastatic disease, PC consults correlated with both hospitalizations and ED visits. Having a hematologic cancer and being unmarried correlated with more hospitalizations and ED visits, respectively. Conclusions: Hospitalizations and ED visits occur in a substantial proportion of cancer CT patients. We need to better understand reasons for these high rates of health care utilization, but the correlations with PC consults suggest that CT patients have unique supportive care needs and that PC services are being targeted to a population particularly in need. [Table: see text]

Abstract 16910: High-Frequency In-Person Visits During Clinical Trial Enrollment is Associated With Relative Reduction in Event Rates in Heart Failure Patients Followed Longitudinally

Circulation ◽  
2020 ◽  
Vol 142 (Suppl_3) ◽  
Author(s):  
Liana C Brooks ◽  
Rohan R Bhat ◽  
Robyn F Farrell ◽  
Mark W Schoenike ◽  
John A Sbarbaro ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
High Frequency ◽  
Trial Participation ◽  
Trial Period ◽  
Single Center ◽  
Hospitalization Rates ◽  
Visit Frequency ◽  
Clinical Trial Enrollment ◽  
Event Rates

Introduction: The COVID-19 Pandemic has mandated limiting routine visit frequency for patients with chronic cardiovascular (CV) diseases. In patients with heart failure (HF) followed longitudinally, the period of clinical trial participation provides an opportunity to evaluate the influence of high-frequency per-protocol in-person visits compared to less frequent routine visits during longitudinal clinical care. Hypothesis: Patients enrolled in clinical trials will have a lower CV and HF event rates during periods of trial enrollment than during non-trial periods. Methods: We examined clinical characteristics, CV and HF hospitalization rates, and outcomes in patients with HF receiving longitudinal HF care at a single center. We evaluated hospitalization rates during the 1-year preceding trial enrollment and hospitalization and death rates during enrollment in clinical trials and for up to 1 year following trial completion. Results: Among the 121 patients enrolled in HF clinical trials, 72% were HFrEF (age 62±11, 19% females, BMI 30.4±6.0, LVEF 25±7, NYHA 2.7±0.6, NT-proBNP 2336±2671) and 28% were HFpEF (age 69±9, BMI 32.1±5.5, 29% females, LVEF 60±10, NYHA 2.4±0.5, NT-proBNP 957±997). Average clinical trial exposure was 8±6.6 months. Per-protocol visit frequency was 16±7 per year during clinical trial enrollment. In the one-year pre-trial period, compared to the within-trial period, CV hospitalizations were 0.88/patient-year vs. 0.32/patient-year (p<0.001) and HF hospitalizations were 0.63/patient-year and 0.24/patient-year (p<0.001), with a mortality rate of 0.04/patient-year during trial participation. In the period of up-to 1 year following the end of trial enrollment CV and HF hospitalizations were intermediate at 0.51/patient-year and 0.27/patient-year with an annualized incremental mortality rate of 0.03/patient-year. Conclusion: In HF patients followed longitudinally at a single center, periods of clinical trial enrollment were associated with high visit frequency and lower CV and HF hospitalization rates. These findings highlight the potential benefits of trial enrollment and high-frequency visits for HF patients at a time when routine visit frequency is being carefully considered during the COVID-19 Pandemic.

Updates in the management and future landscape of urothelial carcinoma

2020 ◽  
pp. 107815522097102
Author(s):  
Kirollos S Hanna ◽  
Maren Campbell ◽  
Adam Kolling ◽  
Alex Husak ◽  
Sabrina Sturm ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Urothelial Carcinoma ◽  
Metastatic Disease ◽  
Checkpoint Inhibitors ◽  
Early Stage ◽  
Locally Advanced ◽  
The United States ◽  
Cancer Type ◽  
Antibody Drug Conjugate ◽  
Early Stage Disease

Urothelial carcinoma is the sixth most common cancer type in the United States. Although most patients present with early stage disease which is associated with improved outcomes, many will progress to locally advanced or metastatic disease. Immune checkpoint inhibitors have significantly impacted the treatment paradigm for patients and have resulted in improved survival rates. Despite their proven efficacy, many ongoing clinical trials continue to refine combinations with chemotherapy, sequencing of therapies and the role of ligand expression. Additionally, novel targets have been identified for advanced urothelial carcinoma and have led to the approval of the antibody-drug conjugate, enfortumab vedotin, and the fibroblast growth factor receptor-targeted, erdafitinib. Enrollment in a clinical trial is strongly encouraged for all stages of advanced or metastatic disease. Numerous ongoing clinical trials are likely to impact the treatment armamentarium for patients. In this manuscript, we highlight key updates in the clinical management for patients and outline ongoing trials.

scholarly journals Influence of Clinical Trial Site Enrollment on Patient Characteristics, Protocol Completion, and End Points

2016 ◽  
Vol 9 (9) ◽  
Author(s):  
Stephen J. Greene ◽  
Adrian F. Hernandez ◽  
Jie-Lena Sun ◽  
Marco Metra ◽  
Javed Butler ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Patient Characteristics ◽  
End Points ◽  
Trial Site ◽  
Clinical Trial Site

Gastric adenocarcinoma treated with radiation with or without epirubicin-based chemotherapy: Evaluation of radiation-induced liver disease.

2011 ◽  
Vol 29 (4_suppl) ◽  
pp. 110-110
Author(s):  
J. D. Schoenfeld ◽  
H. J. Mamon ◽  
L. S. Blaszkowsky ◽  
P. C. Enzinger ◽  
J. Y. Wo ◽  
...  
Keyword(s):  
Liver Disease ◽  
Gastric Adenocarcinoma ◽  
Metastatic Disease ◽  
Liver Toxicity ◽  
Massachusetts General Hospital ◽  
Liver Function Tests ◽  
Liver Change ◽  
Radiation Induced ◽  
Definitive Surgery ◽  
Dose Constraints

110 Background: Anthracycline chemotherapy has been associated with radiation-induced liver disease (RILD). We sought to compare the incidence of liver toxicity among patients with gastric adenocarcinoma treated with radiotherapy (RT) with or without epirubicin-based chemotherapy. Methods: We performed a retrospective analysis of 94 patients with gastric adenocarcinoma treated at Massachusetts General Hospital since November 2005 and the Dana-Farber Cancer Institute since August 1998. All patients underwent definitive surgery and RT (median dose 45 Gray) with a minimum follow up of 6 months. Primary endpoints were development of ascites, radiographic liver change and elevations in liver function tests (LFTs), including alkaline phosphatase (ALP), aspartate transaminase (AST) and alanine transaminase (ALT). Results: In total, 34 patients received epirubicin-based chemotherapy including 9 perioperatively (6 with oxaliplatin and capecitabine [EOX]; 2 with cisplatin and 5-flourouracil [ECF]; 1 with oxaliplatin and 5-flourouracil [EOF]) and 25 postoperatively (2 EOX; 22 ECF; 1 combination). Seven patients were treated with neoadjuvant RT; 87 received adjuvant RT a median of 88 days after surgery (interquartile range 73-108 days). Twenty-one patients developed ascites within 6 months of completing RT, all but one of whom developed peritoneal carcinomatosis or metastatic disease. Among 57 patients that did not develop metastases, maximum elevations in LFTs were similar in patients that received epirubicin-based chemotherapy compared to those who did not (ALP/AST/ALT 150/44/50 vs. 142/41/44, p=0.25/0.36/0.14, respectively), as were rates of radiographic liver change (22% vs. 13%, p=0.44). Conclusions: Epirubicin-based chemotherapy does not significantly increase the risk of RILD in a recent cohort of patients treated with modern RT techniques and dose-constraints. In this setting, treatment of gastric adenocarcinoma with RT and either pre- or postoperative chemotherapy is well tolerated with low rates of liver toxicities. Development of liver toxicity, particularly ascites, within six months of RT may be a harbinger of metastatic disease. No significant financial relationships to disclose.

scholarly journals Patient Characteristics Compete with Dose as Predictors of Acute Treatment Toxicity in Early Phase Clinical Trials

2004 ◽  
Vol 10 (14) ◽  
pp. 4645-4651 ◽  
Author(s):  
André Rogatko ◽  
James S. Babb ◽  
Hao Wang ◽  
Michael J. Slifker ◽  
Gary R. Hudes
Keyword(s):  
Clinical Trials ◽  
Early Phase ◽  
Acute Treatment ◽  
Patient Characteristics ◽  
Treatment Toxicity ◽  
Early Phase Clinical Trials

scholarly journals Patient Characteristics and Temporal Changes in Anticoagulation Treatment Patterns in Patients Diagnosed with Cancer-Associated Thrombosis: An Oscar-US Analysis

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 2132-2132
Author(s):  
Craig I. Coleman ◽  
Kimberly Snow Caroti ◽  
Khaled Abdelgawwad ◽  
George Psaroudakis ◽  
Samuel Fatoba ◽  
...  
Keyword(s):  
Emergency Department ◽  
Metastatic Disease ◽  
Research Funding ◽  
Patient Characteristics ◽  
Treatment Patterns ◽  
Diagnosis Code ◽  
Observation Unit ◽  
Cancer Subtypes ◽  
Cancer Associated Thrombosis ◽  
Active Cancer

Abstract Background: Although guidelines include direct-acting oral anticoagulants (DOACs) as an alternative to low molecular weight heparins (LMWHs) for treatment of cancer associated venous thrombosis (CAT), specific recommendations for selection of patients for DOAC treatment vary. Recommendations for use of DOACs are driven predominantly by perceptions of risk: benefit ratio (often associated with different cancer subtypes). We sought to assess patient characteristics and temporal changes in DOAC (vs. LMWH) utilization in patients being treated in routine practice for CAT. Methods: This analysis is part of the Observational Studies in Cancer Associated Thrombosis for Rivaroxaban - United States (OSCAR-US) program. OSCAR-US is an ongoing study utilizing longitudinal patient-level medical record data from Optum for 91+ million patients seen at 700+ hospitals and 7,000+ clinics across the US. To be included in the present study, adult patients had to be diagnosed with active (primary or metastatic) cancer, undergone hospitalization, emergency department or observation unit admission associated with a primary International Classification of Diseases (ICD)-9th or -10 th revision diagnosis code for venous thromboembolism (VTE) between January 2013 and September 2020, received a DOAC or LMWH on day 7 of CAT treatment, and been active in the data set for at least 12 months prior to the CAT. Cohort assignment was based upon anticoagulant received on day 7 to increase the study's likelihood of appropriately classifying patients into their intended long-term treatment group. We defined active cancer as any cancer associated with ongoing treatment with chemotherapy, immunotherapy, radiation, or recent surgery; the presence of metastatic disease (regardless of time from initial cancer diagnosis); or provider encounter with a primary diagnosis code for cancer within the 6 months prior to the CAT. We excluded patients with an alternative indication for anticoagulation use or evidence of anticoagulation during the 12 months prior (per written prescription or patient self-report). For this analysis, year of CAT diagnosis was grouped into three mutually exclusive categories (2013-2015, 2016-2018, and 2019-2020) to represent early DOAC availability for VTE, peri-DOAC CAT trials and post-CAT guideline recommendation periods. Categorical data were reported as percentages, while continuous data were reported as means ± standard deviations (SDs). Chi-square and independent sample t-tests were used to test for differences in characteristics and treatment patterns between cohorts. Results: In total, 95,072 adult patients experiencing a VTE-related hospitalization, emergency department or observation unit visit were identified. Of these, 14,377 (15.1%) met our pre-specified criteria for active cancer; including 84.7% who received a cancer treatment modality within 6-months and/or had metastatic disease (Table). Though a majority (76.0%) of patients received parenteral therapy as their first anticoagulant upon CAT diagnosis; on day 7 of CAT management, 5325 (52.0%) were receiving a DOAC and 4925 (48.0%) a LMWH. Mean age of patients was 65.2±13.7 years, body mass index (BMI) was 29.9±7.7 kg/m2 and 55.9% were women. Pulmonary embolism (PE)±deep vein thrombosis (DVT) was present in 47.2% of patients and more frequent in patients treated with LMWH than a DOAC on day 7 (p&lt;0.001). The most common cancer types were lung (18.3%) genitourinary (13.5%), breast (12.7%) and colorectal (10.5%) (Figure). The proportion of patients treated with a DOAC increased substantially for all cancers over the three time periods evaluated (27.5% to 55.9% to 73.2%, p&lt;0.001). This temporal relationship of increasing DOAC use (vs. LMWH) remained consistent across individual cancer subtypes (p&lt;0.001 for all). Mean anticoagulant treatment duration was 193±143 days for the entire cohort and was longer in patients treated with DOACs vs. LMWHs (226±138 vs. 147±138 days, p&lt;0.001). Conclusions: In this US population, DOACs were increasingly being utilized for the management of CAT patients and for longer treatment durations than LMWHs. The finding of increasing frequency in use of DOACs vs. LMWHs appeared consistent across all major cancer subtypes. Given their common use, future analyses should evaluate the real-world effectiveness and safety of DOACs compared to LMWHs within individual cancer subtypes. Figure 1 Figure 1. Disclosures Coleman: Bayer AG: Consultancy, Honoraria, Research Funding; Janssen Scientific Affairs LLC: Consultancy, Honoraria, Research Funding; Alexion Pharmaceuticals: Research Funding. Abdelgawwad: Bayer AG: Current Employment. Psaroudakis: Bayer AG: Current Employment. Fatoba: Bayer AG: Current Employment. Rivera: Bayer AG: Current Employment. Brobert: Bayer AG: Current Employment.

Informed consent for early-phase clinical trials: therapeutic misestimation, unrealistic optimism and appreciation

2019 ◽  
Vol 45 (6) ◽  
pp. 384-387 ◽  
Author(s):  
Jodi Halpern ◽  
David Paolo ◽  
Andrew Huang
Keyword(s):  
Clinical Trials ◽  
Informed Consent ◽  
Early Phase ◽  
Phase 1 ◽  
Trial Participation ◽  
Unrealistic Optimism ◽  
Personal Significance ◽  
Decision Making Capacity ◽  
Early Phase Clinical Trials ◽  
Motivation To Participate

Unrealistic therapeutic beliefs are very common—the majority of patient-subjects (up to 94%) enrol in phase 1 trials seeking and expecting significant medical benefit, even though the likelihood of such benefit has historically proven very low. The high prevalence of therapeutic misestimation and unrealistic optimism in particular has stimulated debate about whether unrealistic therapeutic beliefs in early-phase clinical trials preclude adequate informed consent. We seek here to help resolve this controversy by showing that a crucial determination of when such therapeutic beliefs are ethically problematic turns on whether they are causally linked and instrumental to the motivation to participate in the trial. Thus, in practice, it is ethically incumbent on researchers to determine which understanding and beliefs lead to the participant’s primary motivation for enrolling, not to simply assess understanding, beliefs and motivations independently. We further contend that assessing patient-subjects’ appreciation as a component of informed consent—it is already an established component of decision-making capacity assessments—can help elucidate the link between understanding-beliefs and motivation; appreciation refers to an individual’s understanding of the personal significance of both the medical facts and the experience of trial participation. Therefore, we recommend that: (1) in addition to the usual question, ‘Why do you want to participate in this trial?’, all potential participants should be asked the question: ‘What are you giving up by participating in this trial?’ and (2) researchers should consider the settings in which it may be possible and practical to obtain ‘two-point consent’.

Clinical response to imatinib mesylate in recurrent and metastatic gastrointestinal stromal tumors

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 20528-20528
Author(s):  
R. Villalobos ◽  
G. Hernández ◽  
M. Martinez-Prieto ◽  
A. Silva
Keyword(s):  
Imatinib Mesylate ◽  
Metastatic Disease ◽  
Gastrointestinal Stromal Tumors ◽  
Patient Characteristics ◽  
Systemic Hypertension ◽  
Unknown Primary ◽  
Stromal Tumors ◽  
Vein Thrombosis ◽  
Drug Intolerance ◽  
The Moment

20528 Background: We analyzed the efficacy and safety of imatinib mesylate in GIST (Gastrointestinal Stromal Tumors) in various anatomic sites. Methods: Out of 24 patients with recurrent or metastatic disease receiving treatment, clinical and tomographic evaluation was possible only in 21 pts. RECIST criteria were used to evaluate response and NCI guidelines were used for evaluation of toxicity. The treatment given between March 2003 and December 2006 was imatinib mesylate 200mg twice daily until disease progression or drug intolerance. All included patients had histopathologic confirmation and CD 117 positive inmunohistochemistry. Results: Patient characteristics were as follows: 19 females, 2 males, average age: 37.8 years (range: 36 - 80), Functional status according to ECOG: 0 in 19 pts, 1 in one patient and 3 in one patient. Anatomic sites were: 4 stomach, 7 small intestine, 3 rectum-sigmoid, 4 carcinomatosis from unknown primary, 2 peripancreatic and one esophageal. Nine patients had recurrent disease, 12 unresectable disease and 11 pts had liver metastases and 13 had retroperitoneal metastatic disease. Comorbidities: 3 pts with Type 2 Diabetes Mellitus one patient with Systemic Hypertension, and deep vein thrombosis each, two patients with a previous diagnosis of cancer: Thyroid and Lung Cancer respectively. Responses:2 (9.5%)CR, 6 (28.5%) PR, 12 (57.1%) stable disease, one (4.7%) pt. with progressive disease. Toxicity: one patient had rash, one patient presented bone pain, one patient with face discoloration, one with cephalea and nausea, and two patients with bipalpebral edema. Median duration for response was 5.7 months (range 2–30) at the moment. Conclusions: Efficacy of imatinib mesylate in recurrent and metastatic GIST's is high with overall response rates of 95% and adequate tolerance. Studies as adyuvant and second line therapy are needed in our population. No significant financial relationships to disclose.

A phase II study of trastuzumab-DM1 (T-DM1), a HER2 antibody-drug conjugate (ADC), in patients (pts) with HER2+ metastatic breast cancer (MBC): Final results

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 1017-1017
Author(s):  
C. L. Vogel ◽  
H. A. Burris ◽  
S. Limentani ◽  
R. Borson ◽  
J. O'Shaughnessy ◽  
...  
Keyword(s):  
Phase Ii ◽  
Metastatic Disease ◽  
Targeted Delivery ◽  
Phase Ii Study ◽  
Single Agent ◽  
Objective Response ◽  
Metastatic Breast ◽  
Antibody Drug Conjugate ◽  
Metastatic Setting

1017 Background: T-DM1 is an ADC that combines the biological activity of trastuzumab (T) with targeted delivery of a potent antimicrotubule agent, DM1, to HER2-expressing cancer cells. In a phase I study, T-DM1 was administered IV q3w to pts with HER2+ MBC who had progressed on T + chemotherapy. T-DM1 was well-tolerated at the maximum tolerated dose (MTD) of 3.6 mg/kg, with no reports of cardiac toxicity. The confirmed objective response rate (ORR) for the 9 pts with measurable disease treated at the MTD was 44%. The phase II study described here further assesses tolerability and activity of T-DM1. Methods: This was a multi-institutional, open-label, single-arm phase II study, to enroll 100 pts. All eligible pts had progressed on HER2-directed therapy and had received chemotherapy in the metastatic setting. T-DM1 was administered at 3.6 mg/kg IV q3w. Primary objectives were assessment of ORR and of safety and tolerability. Results: As of the August 29, 2008, data-cut, 112 patients had enrolled, with baseline median age 54.5 (range 33–82); ECOG PS 2 or 3, 80%; 68.7% with > 3 sites of metastatic disease; median 3 (range 1–14) prior chemotherapy agents for metastatic disease, median 76.3 weeks prior T, and 55.4% with previous lapatinib. Due to limited F/U, the median number of T-DM1 cycles received was 5 (range 1–16), and 19 of the 107 efficacy evaluable patients had only one post-baseline tumor assessment. Fifty-six pts had discontinued study treatment. With a median follow-up of 4.4 mos, there were 42 (39.3%) ORs (CR or PR), 29 (27.1%) of which have been confirmed by follow-up (F/U) imaging. Among the subgroup of pts who had either >6 months F/U or had discontinued from the study at any time (n = 76) there were 33 ORs (43.4%), 29 (38.2%) of which were confirmed by F/U imaging. The most common grade 3–4 AE was thrombocytopenia (7.1%). Updated data will be presented at the meeting, including updated ORR, 6-month clinical benefit rate, ORR by independent review, progression-free survival, and duration of response. Conclusions: T-DM1 has single-agent activity in pts with previously treated, HER2+ MBC, and is well tolerated at the recommended phase II dose. [Table: see text]

Sign in / Sign up

Export Citation Format

Share Document