Use of real-world clinical lab data to reveal Asian NTRK fusion test availability patterns.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e15574-e15574
Author(s):  
Markus Eckstein ◽  
Kenneth Joel Bloom ◽  
Susanne Munksted Fosvig ◽  
Marieke Hoefsmit ◽  
Jordan Clark
Keyword(s):  
Real World ◽  
First Generation ◽  
Treatment Options ◽  
Laboratory Data ◽  
Clinical Pathology ◽  
Pathology Laboratory ◽  
Pediatric Cancer Patients ◽  
The Us ◽  
Trk Inhibitors ◽  
The Impact

e15574 Background: The development and launch of first-generation tropomyosin receptor kinase (TRK) inhibitors, such as larotrectinib and entrectinib, has brought targeted treatment options to neurotrophic tropomyosin-related kinase (NTRK) fusion–positive adult and pediatric cancer patients who may have been out of treatment options. Widespread adoption of NTRK fusion testing is needed to support successful selection of patients for these TRK inhibitors as well as other targeted therapies. The objective of this study was to investigate global availability of NTRK fusion testing, with a specific focus on Asia. Methods: For this study, real-world clinical pathology laboratory data, derived from Diaceutics’ proprietary Global Diagnostic Index (GDI) of more than 2500 labs, were investigated to assess the current footprint of NTRK fusion testing in Asia. Availability of clinical routine testing was analyzed in selected cohorts of laboratories across different regions, comparing Asia to the US and Europe. Laboratories were included based on their large volume of testing or on their importance (e.g., large academic and large commercial laboratories). Results: Data on availability of NTRK fusion testing by clinical laboratories reveal different patterns of NTRK fusion test adoption in Asia, depending on the region under study. In China and similarly in the US, Italy, and France clinical NTRK fusion testing is already being performed by the majority of laboratories from the selected cohort. In Japan, physicians have limited access to testing, with only a few laboratories from the selected cohort performing testing for NTRK fusions. Conclusions: NTRK fusion testing is essential for identifying patients eligible for first-generation TRK-inhibitors. Various laboratories across selected cohorts of labs in China and Japan lack the capability to initiate clinical NTRK fusion testing in-house, denying some patients the potential benefit from these breakthrough therapies. Consequently, patient outcomes will be suboptimal due to lack of testing infrastructure. The readiness of Asian markets to support patient selection successfully can affect the rate of testing and the impact of these innovative therapies on patients’ lives. [Table: see text]

scholarly journals Abuse-deterrent opioid analgesics: a guide for clinicians

2020 ◽  
Vol 10 (1) ◽  
pp. 55-62 ◽  
Author(s):  
Adam J Carinci
Keyword(s):  
Real World ◽  
Opioid Analgesics ◽  
Prescription Opioid ◽  
Pharmacokinetic Studies ◽  
Regulatory Issues ◽  
The Us ◽  
Us Fda ◽  
The Impact ◽  
Prescription Opioid Abuse

The US FDA has encouraged the development of abuse-deterrent formulations (ADFs) of opioid analgesics as one component in a comprehensive effort to combat prescription opioid abuse. Guidance issued by the FDA outlines three types of premarket studies for evaluating abuse deterrence: laboratory-based in vitro manipulation and extraction studies, pharmacokinetic studies and human abuse potential studies. After approval, postmarket studies are needed to evaluate the impact of an ADF product on abuse in real-world settings. This review summarizes the regulatory issues involved in the development of ADF opioids and clarifies abuse-deterrence claims in product labels, in order to assist clinicians in critically evaluating the available evidence pertaining to the abuse-deterrent features of opioid analgesics.

scholarly journals Caregiver-Reported Burden in RE-KINECT: Data From a Prospective Real-World Tardive Dyskinesia Screening Study

2021 ◽  
pp. 107839032110235
Author(s):  
Andrew J. Cutler ◽  
Stanley N. Caroff ◽  
Caroline M. Tanner ◽  
Huda Shalhoub ◽  
William R. Lenderking ◽  
...  
Keyword(s):  
Tardive Dyskinesia ◽  
Real World ◽  
Treatment Options ◽  
Body Region ◽  
Full Time ◽  
Abnormal Involuntary Movements ◽  
Involuntary Movements ◽  
Screening Study ◽  
Part Time ◽  
The Impact

Background: RE-KINECT (NCT03062033), a real-world study of possible tardive dyskinesia (TD) in antipsychotic-treated patients, included a questionnaire to assess the effects of patients’ abnormal involuntary movements on caregivers. Objective: To capture the experiences of caregivers who assisted individuals with abnormal involuntary movements that were confirmed by clinicians as being consistent with TD. Methods: Qualified (nonpaid) caregivers were invited to complete a questionnaire that included the following: caregivers’ sociodemographic characteristics, their perceptions about the impact of abnormal involuntary movements on patients, and the impact of these movements on themselves (caregivers). Results: Of the 41 participating caregivers, 25 (61.0%) were women, 20 (48.8%) were employed full time or part time, and 35 (85.4%) were family members or friends. Based on responses from caregivers who noticed patients’ abnormal involuntary movements and were caring for individuals who also noticed those movements, 48.0% of patients had “a lot” of severity in ≥1 body region and 76.0% had abnormal involuntary movements in ≥2 regions. Caregiver ratings were significantly correlated with patient ratings (but not with clinician ratings) for maximum severity of abnormal involuntary movements and the number of affected regions (both p <.05). Based on their own judgments and perceptions, caregivers reported that the patient’s movements had “some” or “a lot” of impact on their (caregiver’s) ability to continue usual activities (50.0%), be productive (58.3%), socialize (55.6%), or take care of self (50.0%). Conclusion: Caregivers as well as patients are negatively affected by TD, and the impact of TD on caregivers’ lives should be considered when determining treatment options.

scholarly journals The Association of Real-World CA 19-9 Level Monitoring Patterns and Clinical Outcomes Among Patients With Metastatic Pancreatic Ductal Adenocarcinoma

2021 ◽  
Vol 11 ◽  
Author(s):  
Ben George ◽  
Matthew Kent ◽  
Andy Surinach ◽  
Neil Lamarre ◽  
Paul Cockrum
Keyword(s):  
Clinical Outcomes ◽  
Real World ◽  
Treatment Initiation ◽  
Population Based ◽  
Ductal Adenocarcinoma ◽  
Prognostic Impact ◽  
Testing Frequency ◽  
The Us ◽  
The Impact ◽  
Level Monitoring

BackgroundPancreatic cancer is expected to be the third deadliest cancer in the US in 2021. Evaluation of treatment response in patients with mPDAC necessitates scheduled clinical and radiographic assessments along with monitoring serum CA 19-9 levels. Currently available single-institution data examining the importance of CA 19-9 monitoring cannot be generalized to real-world settings. We investigated the impact of serum CA 19-9 monitoring and its association with clinical outcomes in patients with mPDAC in a population-based setting.MethodsData were extracted from the Flatiron Health electronic health record (EHR)-derived de-identified database for patients diagnosed with mPDAC between January 1, 2015, and June 30, 2020. Serum CA 19-9 levels at baseline – defined as the values obtained ≤ 60 days prior to treatment initiation - and during treatment were extracted. CA 19-9 levels &gt; 40 IU/mL were considered elevated. Survival outcomes were compared based on testing frequency, baseline CA 19-9 levels, and change in CA 19-9.Results6,118 patients with mPDAC who received treatment were included in the analysis. The median age at diagnosis was 68 years (IQR: 61-75). Patients with normal baseline CA 19-9 experienced longer median survival than patients with elevated levels [1L: 8.8 months (95% CI: 7.9 - 10) vs. 7.2 months (6.8 – 7.5), p &lt; 0.001; 2L: 7.2 months (6.1 – 9.2) vs. 5.2 months (4.9 – 5.6), p &lt; 0.001; 3L: 6.1 months (5.4 – 9.1) vs. 3.9 months (3.4 – 4.3), p &lt; 0.001]. Patients with decreasing/stable CA 19-9 during treatment experienced longer survival than patients who experienced an increase in CA 19-9 levels [1L: 10.9 months (10.5 – 11.3) vs. 5.4 months (5.1 – 5.9), p &lt; 0.0001; 2L: 8.2 months (7.7 – 8.5) vs. 4.3 months (4.1 – 4.7), p &lt; 0.001; 3L: 7.5 months (6.6 – 9.2) vs. 3.7 months (3.4 – 4.3), p &lt; 0.001].ConclusionsIn one of the largest, contemporary, real-world studies of patients with mPDAC, elevated CA 19-9 level at treatment initiation demonstrated a prognostic impact. Routine serial monitoring of CA 19-9 levels during treatment may be warranted, in addition to clinical and radiographic assessment, and may translate into better patient outcomes. Further validation studies are needed to understand the generalizability of these results.

scholarly journals Real-world experience with pasireotide-LAR in resistant acromegaly: a single center 1-year observation

Pituitary ◽  
2021 ◽  
Author(s):  
Maria Stelmachowska-Banaś ◽  
Izabella Czajka-Oraniec ◽  
Agnieszka Tomasik ◽  
Wojciech Zgliczyński
Keyword(s):  
Glucose Metabolism ◽  
Real World ◽  
First Generation ◽  
Somatostatin Receptor ◽  
Real Life ◽  
Pituitary Surgery ◽  
Complete Response ◽  
Single Center ◽  
Life Study ◽  
The Impact

Abstract Context Pasireotide-LAR, a second-generation somatostatin receptor ligand (SRL), is recommended for patients with acromegaly as second-line treatment. Its efficacy and safety were assessed in clinical trials; however, the real-world evidence is still scarce. Objective The aim of this study was to evaluate the impact of 1-year treatment with pasireotide-LAR on disease control and glucose metabolism in acromegaly patients resistant to first-generation SRLs. Design A single-center prospective study. Methods Twenty-eight patients with active acromegaly or acrogigantism on first-generation SRLs following ineffective pituitary surgery were switched to treatment with pasireotide-LAR 40 or 60 mg i.m. every 28 days. To assess the efficacy of the treatment GH and IGF-1 levels were measured every 3 months. Safety of treatment was carefully evaluated, especially its impact on glucose metabolism. Results Complete biochemical control (GH ≤ 1 ng/mL and IGF-1 ≤ 1 × ULN) was achieved in 26.9% of patients and partial + complete response (GH ≤ 2.5 ng/mL and IGF-1 ≤ 1.3 × ULN) in 50.0% of patients. Mean GH level decrease was the largest within first 6 months (P = 0.0001) and mean IGF-1 level decreased rapidly within the first 3 months (P < 0.0001) and they remained reduced during the study. Blood glucose and HbA1c levels increased significantly within 3 months (P = 0.0001) and stayed on stable level thereafter. Otherwise, the treatment was well tolerated and clinical improvement was noticed in majority of patients. Conclusions This real-life study confirmed good effectiveness of pasireotide-LAR in patients resistant to first-generation SRLs. Pasireotide-LAR was overall safe and well tolerated, however significant glucose metabolism worsening was noted.

scholarly journals 571. “Real world” Impact of Letermovir for Prevention of Cytomegalovirus Infection in Hematopoietic-Cell Transplantation

2020 ◽  
Vol 7 (Supplement_1) ◽  
pp. S351-S351
Author(s):  
Alyssa M Loecher ◽  
Kendra Yum ◽  
Daniel Park ◽  
Sara Kim ◽  
Patricia Saunders-Hao ◽  
...  
Keyword(s):  
Antiviral Therapy ◽  
Median Duration ◽  
Real World ◽  
Laboratory Data ◽  
Hematopoietic Cell ◽  
Cell Transplant ◽  
The Real ◽  
Allogeneic Hct ◽  
Significant Difference ◽  
The Impact

Abstract Background Letermovir (LTM) was FDA-approved in 2017 for cytomegalovirus (CMV) prophylaxis in allogeneic hematopoietic cell transplant (HCT) patients. We evaluated the “real-world” impact of LTM in adult HCT recipients at the Mount Sinai Hospital in New York following addition of LTM to our formulary in June 2018. Methods In this retrospective study, we compared 31 consecutive allogeneic HCT recipients from June 2018-June 2019 who received LTM prophylaxis from Day +10–100 to 36 consecutive CMV-seropositive patients who underwent allogeneic HCT June 2017-June 2018 and did not receive LTM. Clinical and laboratory data were abstracted from the existing electronic medical record. The primary outcome was development of clinically significant CMV infection (CS-CMV) through 6 months post-HCT, defined as CMV viremia or disease requiring antiviral therapy. Results There were no baseline differences in patient demographics or transplant characteristics between patients who did and did not receive LTM prophylaxis. The median duration of LTM prophylaxis was 96 days (IQR, 66–116). At 6-months post-HCT, the cumulative incidence of CS-CMV was lower in patients receiving LTM prophylaxis vs no prophylaxis (29% vs 61%, respectively; p=0.004, Figure 1). Patients who received LTM developed CS-CMV at a later time post-HCT than those who did not receive prophylaxis (median 160 vs 39 days, respectively; p &lt; 0.01). Among patients with CS-CMV, the median duration of antiviral therapy was 39 vs 70 days in the LTM prophylaxis vs no prophylaxis groups, respectively (p&lt; 0.01). While there was no difference in the incidence of graft-versus-host disease (GVHD), the median time to GVHD diagnosis was longer in the LTM group (63 days vs 27 days in the no prophylaxis group; p=0.02). Between patients who did and did not receive LTM prophylaxis no statistically significant difference was observed, respectively, in incidence of CMV disease (3% vs 14%), hospital re-admission (68% vs 67%), or mortality (26% vs 14%) at 6 months post-HCT. Conclusion We demonstrated the “real-world” utility of LTM for CMV prevention in HCT recipients. Future studies should further evaluate the impact of LTM on antiviral drug usage, GVHD, and other transplant-related outcomes in this population. Disclosures All Authors: No reported disclosures

Surgery for Lung Cancer and the Consequences for the Swallow

2016 ◽  
Vol 1 (13) ◽  
pp. 162-168
Author(s):  
Pippa Hales ◽  
Corinne Mossey-Gaston
Keyword(s):  
Lung Cancer ◽  
Treatment Options ◽  
Vocal Cord Palsy ◽  
Subsequent Treatment ◽  
Physiological Reserve ◽  
Palliative Phase ◽  
And Function ◽  
The Impact ◽  
Swallow Function

Lung cancer is one of the most commonly diagnosed cancers across Northern America and Europe. Treatment options offered are dependent on the type of cancer, the location of the tumor, the staging, and the overall health of the person. When surgery for lung cancer is offered, difficulty swallowing is a potential complication that can have several influencing factors. Surgical interaction with the recurrent laryngeal nerve (RLN) can lead to unilateral vocal cord palsy, altering swallow function and safety. Understanding whether the RLN has been preserved, damaged, or sacrificed is integral to understanding the effect on the swallow and the subsequent treatment options available. There is also the risk of post-surgical reduction of physiological reserve, which can reduce the strength and function of the swallow in addition to any surgery specific complications. As lung cancer has a limited prognosis, the clinician must also factor in the palliative phase, as this can further increase the burden of an already compromised swallow. By understanding the surgery and the implications this may have for the swallow, there is the potential to reduce the impact of post-surgical complications and so improve quality of life (QOL) for people with lung cancer.

Rapid evaluation of FDG imaging alternatives using head-to-head comparisons of full ring and gamma camera based PET scanners –

2002 ◽  
Vol 41 (05) ◽  
pp. 208-213 ◽  
Author(s):  
L. M. Haslinghuis-Bajan ◽  
L. Hooft ◽  
A. van Lingen ◽  
M. van Tulder ◽  
W. Devillé ◽  
...  
Keyword(s):  
Methodological Quality ◽  
Gamma Camera ◽  
First Generation ◽  
Prospective Evaluation ◽  
First Approximation ◽  
Variable Head ◽  
Malignant Lesions ◽  
The Impact ◽  
Pet Scanners

SummaryAim: While FDG full ring PET (FRPET) has been gradually accepted in oncology, the role of the cheaper gamma camera based alternatives (GCPET) is less clear. Since technology is evolving rapidly, “tracker trials” would be most helpful to provide a first approximation of the relative merits of these alternatives. As difference in scanner sensitivity is the key variable, head-to-head comparison with FRPET is an attractive study design. This systematic review summarises such studies. Methods: Nine studies were identified until July 1, 2000. Two observers assessed the methodological quality (Cochrane criteria), and extracted data. Results: The studies comprised a variety of tumours and indications. The reported GC- and FRPET agreement for detection of malignant lesions ranged from 55 to 100%, but with methodological limitations (blinding, standardisation, limited patient spectrum). Mean lesion diameter was 2.9 cm (SD 1.8), with only about 20% <1.5 cm. The 3 studies with the highest quality reported concordances of 74-79%, for the studied lesion spectrum. Contrast at GCPET was lower than that of FRPET, contrast and detection agreement were positively related. Logistic regression analysis suggested that pre-test indicators might be used to predict FRPET-GCPET concordance. Conclusion: In spite of methodological limitations, “first generation” GCPET devices detected sufficient FRPET positive lesions to allow prospective evaluation in clinical situations where the impact of FRPET is not confined to detection of small lesions (<1.5 cm). The efficiency of head-to-head comparative studies would benefit from application in a clinically relevant patient spectrum, with proper blinding and standardisation of acquisition procedures.

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