scholarly journals Real‐world treatment patterns, adverse events and clinical outcomes in patients with chronic lymphocytic leukaemia treated with ibrutinib in the UK

eJHaem ◽  
2021 ◽  
Author(s):  
Peter Hillmen ◽  
Jing Xie ◽  
Alan S. M. Yong ◽  
Catherine Waweru ◽  
Thuy Anh Sorof ◽  
...  
Keyword(s):  
Adverse Events ◽  
Chronic Lymphocytic Leukaemia ◽  
Clinical Outcomes ◽  
Real World ◽  
Lymphocytic Leukaemia ◽  
Treatment Patterns ◽  
The Uk

scholarly journals A platform trial in practice: adding a new experimental research arm to the ongoing confirmatory FLAIR trial in chronic lymphocytic leukaemia

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Dena R. Howard ◽  
Anna Hockaday ◽  
Julia M. Brown ◽  
Walter M. Gregory ◽  
Susan Todd ◽  
...  
Keyword(s):  
Chronic Lymphocytic Leukaemia ◽  
Type I Error ◽  
Lymphocytic Leukaemia ◽  
Experimental Therapy ◽  
Type I ◽  
Open Label ◽  
Additional Funding ◽  
Trial Structure ◽  
Randomised Controlled ◽  
The Uk

Abstract Background The FLAIR trial in chronic lymphocytic leukaemia has a randomised, controlled, open-label, confirmatory, platform design. FLAIR was successfully amended to include an emerging promising experimental therapy to expedite its assessment, greatly reducing the time to reach the primary outcome compared to running a separate trial and without compromising the validity of the research or the ability to recruit to the trial and report the outcomes. The methodological and practical issues are presented, describing how they were addressed to ensure the amendment was a success. Methods FLAIR was designed as a two-arm trial requiring 754 patients. In stage 2, two new arms were added: a new experimental arm and a second control arm to protect the trial in case of a change in practice. In stage 3, the original experimental arm was closed as its planned recruitment target was reached. In total, 1516 participants will be randomised to the trial. Results The changes to the protocol and randomisation to add and stop arms were made seamlessly without pausing recruitment. The statistical considerations to ensure the results for the original and new hypotheses are unbiased were approved following peer review by oversight committees, Cancer Research UK, ethical and regulatory committees and pharmaceutical partners. These included the use of concurrent comparators in case of any stage effect, appropriate control of the type I error rate and consideration of analysis methods across trial stages. The operational aspects of successfully implementing the amendments are described, including gaining approvals and additional funding, data management requirements and implementation at centres. Conclusions FLAIR is an exemplar of how an emerging experimental therapy can be assessed within an existing trial structure without compromising the conduct, reporting or validity of the trial. This strategy offered considerable resource savings and allowed the new experimental therapy to be assessed within a confirmatory trial in the UK years earlier than would have otherwise been possible. Despite the clear efficiencies, treatment arms are rarely added to ongoing trials in practice. This paper demonstrates how this strategy is acceptable, feasible and beneficial to patients and the wider research community. Trial registration ISRCTN Registry ISRCTN01844152. Registered on August 08, 2014

CTNI-59. A MULTICENTER OBSERVATIONAL STUDY OF CS-131 SEEDS EMBEDDED IN A COLLAGEN CARRIER TILE FOR NEWLY DIAGNOSED AND RECURRENT OPERABLE INTRACRANIAL NEOPLASMS – TRIAL IN PROGRESS

2021 ◽  
Vol 23 (Supplement_6) ◽  
pp. vi74-vi74
Author(s):  
Erin Dunbar ◽  
David McCracken ◽  
adam nowlan ◽  
Clark Chen ◽  
Kathryn Dusenbery ◽  
...  
Keyword(s):  
Overall Survival ◽  
Radiation Therapy ◽  
Adverse Events ◽  
Clinical Outcomes ◽  
Real World ◽  
External Beam Radiation ◽  
Newly Diagnosed ◽  
Intracranial Neoplasms ◽  
Safety And Efficacy ◽  
Patient Reported

Abstract BACKGROUND For patients with operable intracranial neoplasms, there are opportunities to augment local control beyond traditional methods, such as external beam radiation therapy. Brachytherapy, the implantation of radioactive sources into the resection cavity, can be useful in this setting by providing immediate initiation of radiation and limiting the exposure of surrounding normal tissue to radiation. Traditional intracranial brachytherapy has been limited by uneven dose distributions, complicated workflows, extended procedural times, cost of dedicated equipment, and frequent adverse events. To address these issues, a permanently implanted device with Cs-131 radiation seeds embedded in a bioresorbable collagen carrier tile (GammaTile, GT Medical Technologies, Tempe, AZ USA) was developed. Described as surgically targeted radiation therapy (STaRT), it is FDA-cleared for use in newly-diagnosed malignant intracranial neoplasms and recurrent intracranial tumors, and has demonstrated excellent safety and efficacy in early commercial use. The primary objectives of this multicenter, prospective, observational (phase IV) registry study are to evaluate “real-world” clinical outcomes and patient-reported outcomes that measure the safety and efficacy of STaRT using the GammaTile. METHODS Patients undergoing resection (R) of brain tumors with intra-operative GammaTile placement are eligible for enrollment. Planned sample size is 600 at up to 50 enrolling sites. First subject was enrolled 10/14/2020. Tumor pathology, overall survival, radiation- and surgery-related adverse events, patient- and provider-reported quality of life, serial MRIs, and timing of surgical bed and/or distant recurrence are collected. Powered primary endpoints for recurrent brain metastases, recurrent glioblastoma, and recurrent meningioma (surgical bed-progression free survival (PFS), overall survival, and PFS, respectively), compare STaRT to standard-of-care benchmarks. Results will be used to improve awareness and access to this treatment, benchmark clinical outcomes in the real-world setting, allow for comparisons to existing treatments, facilitate the design of future clinical trials, and contribute to the optimal sequencing of treatments for intracranial neoplasms.

Treatment practices, response to therapy and adverse events in ANCA-associated vasculitis patients in Europe: top-line findings from a retrospective observational study

2019 ◽  
Author(s):  
Peter Rutherford ◽  
Dieter Goette
Keyword(s):  
Adverse Events ◽  
Clinical Outcomes ◽  
Real World ◽  
Response To Therapy ◽  
Remission Induction ◽  
Induction Treatment ◽  
Symptom Duration ◽  
Similar Distribution ◽  
Presenting Symptoms ◽  
Anca Associated Vasculitis

Abstract Background ANCA-associated vasculitis patient outcome data in the real world setting is scarce. This study measures key clinical outcomes and adverse effects over the first 12 months of remission induction therapy.Methods This was a retrospective study of 929 newly diagnosed [ND] and 268 relapsing patients [RP] conducted online by 399 clinicians. Each clinician completed a survey for 3 patients meeting the following criteria: initiated remission induction treatment for new or relapsing disease between Nov 2014 and Feb 2017, ≥ 6 months of therapy including ≥ 1 course of induction therapy, under continuous care for ≥12 months. Data were collected relating to baseline presentation and at 1, 3, 6, and 12 months.Results 58% were >55 years old with more granulomatosis with polyangiitis (GPA, 54%) versus microscopic polyangiitis (MPA, 46%), and <20% of patients had Birmingham Vasculitis Activity Scoring (BVAS) performed. Median symptom duration prior to diagnosis was 6 to 7 weeks. Presenting symptoms were similar between ND and RP, noted differences (≥ 5%) were more fever, rash, and neuropathy, and less renal disease in RP. The majority (68% ND and 84% RP) had at least one comorbidity at diagnosis, with a similar distribution. Glucocorticoids (GC) were used by 83% ND and 76% RP; >50% were still receiving GC at 12 months. Most common treatments were cyclophosphamide+GC for ND (59%) and rituximab+GC for RP (44%). Many patients had slow and/or partial response to therapy, by 12 months >60% had a full response. 81% of patients with response by month 1 maintained full response through month 12. Adverse events and infections were common, especially during the first 3 months when GC use is highest.Conclusions Real world data show that both ND and RP ANCA-associated vasculitis patients respond variably to induction remission treatment and many experience adverse events and infections over the first 12 months of treatment. The presence of comorbidities at treatment initiation in most patients compounded the adverse impacts of disease and treatment. This study improves our understanding of the reality of clinical outcomes in ANCA-associated vasculitis and the need for targeted therapeutic approaches.

scholarly journals Treatment patterns and clinical outcomes with palbociclib-based therapy received in US community oncology practices

2020 ◽  
Author(s):  
Junji Lin ◽  
Lynn McRoy ◽  
Maxine D Fisher ◽  
Nan Hu ◽  
Cralen Davis ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Clinical Outcomes ◽  
Real World ◽  
Medical Records ◽  
Metastatic Breast ◽  
Patient Characteristics ◽  
Treatment Patterns ◽  
Community Oncology ◽  
Label Indication ◽  
Free Rate

Background: Limited studies have evaluated palbociclib-based therapy use in patients with advanced/metastatic breast cancer in the real world. This retrospective study used medical records from US community oncology practices to address the gap. Materials & methods: Eligible patients receiving palbociclib-based therapy per label indication from 3 February 2015 to 31 December 2017 were included. Descriptive analyses were conducted for patient characteristics, treatment patterns and clinical outcomes. Results: The study included 233 patients who received palbociclib + aromatase inhibitor (P+AI) and 48 who received palbociclib + fulvestrant (P+F). Real-world progression-free rate for P+AI was 69.8% (46.8%) at 12 (24) months (P+F: 43.5% [39.9%]) months. Real-world survival rate was 89.8% (71.4%) at 12 (24) months (P+F: 76.3% [65.0%]). Conclusion: The study findings are consistent with previous studies of palbociclib-based therapy.

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