scholarly journals An Observational Study of Fostamatinib As Second Line Therapy in Adult Patients with Immune Thrombocytopenia (ITP) in Real-World Clinical Practice

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4223-4223
Author(s):  
David Hughes ◽  
Robert P. Numerof ◽  
Andrea Zider ◽  
Donna Chow ◽  
Ruchika Goel
Keyword(s):  
Quality Of Life ◽  
Platelet Count ◽  
Real World ◽  
Adult Patients ◽  
Second Line ◽  
Platelet Response ◽  
Publicly Traded ◽  
Second Line Therapy ◽  
Line Therapy

Abstract Background: In immune thrombocytopenia (ITP), autoantibody-bound platelets are targeted for phagocytosis by Fcγ-receptors on macrophages in a spleen tyrosine kinase (SYK)-dependent signaling pathway. Fostamatinib is a potent oral SYK inhibitor that demonstrated efficacy for the treatment of ITP in two phase 3 randomized, placebo-controlled, double-blind studies and long-term durable responses in the phase 3 open-label extension study. In these studies, 146 patients with ITP were treated with fostamatinib, and 54% had a platelet response (platelet count ≥50,000/µL), which was maintained over a median of 86% of treatment days. These studies led to approval of fostamatinib in the US, Europe, and Canada as a therapy for chronic ITP in adult patients who have had an insufficient response to a prior treatment. Further, a post-hoc analysis showed that, in those who received fostamatinib as second-line therapy (following steroids ± immunoglobulins), 78% achieved a platelet response, which was maintained over a median of 83% of treatment days. 1 Adverse events (AEs) were reported in 72% of second-line patients and 86% of all patients, and common AEs were similar between second-line patients and the total patient population. 1, 2 Bleeding events were reported in 24% of second-line patients vs. 41% of all patients. The results of the post hoc analysis underscored the necessity for further evaluation of fostamatinib as a second-line therapy for ITP. We are conducting a multi-center, prospective, observational, two cohort study of fostamatinib as second-line therapy for the management of ITP in adult patients who have an insufficient response to prior steroids ± immunoglobulins. Aim: This study will evaluate clinical outcomes and safety of fostamatinib as second-line therapy for ITP in adult patients in real-world clinical practice. Patients will also be assessed for quality of life and utilization of health care resources during the study. Methods: The study (FORTE; NCT04904276; O-FOSTA-901) is being conducted at 10 sites across the US and will enroll 45 patients with ITP into 2 cohorts (see Figure). The study is registered at https://clinicaltrials.gov/ct2/show/NCT04904276. The study aims to enroll adult patients with a confirmed diagnosis of ITP and an inadequate response to prior steroids ± immunoglobulins. Patients who have received any prior ITP treatment other than steroids or immunoglobulins will be excluded. Patients who will initiate fostamatinib as second-line therapy at study entry will be enrolled into Cohort 1; patients currently receiving fostamatinib as second-line therapy with the intent to continue treatment will be enrolled in Cohort 2 (historical platelet counts will be retrospectively collected). Patients will be treated by their clinician at the clinician's discretion (not pre-specified by a study protocol). Primary outcome measures will include platelet count over time, duration of platelet count elevation, use of ITP rescue medication, changes in dosing, use of concomitant ITP medications, incidence of serious and ITP-related AEs, and quality of life. Quality of life assessments will utilize the SF-36v2 and ITP-PAQ. All data will be collected at regularly scheduled clinic visits according to the clinician's normal practice for 12 months or until fostamatinib is discontinued. Summary: This ongoing observational study of adult patients with ITP will enable further characterization of the risk/benefit profile of fostamatinib as a second-line treatment for ITP and will provide insight on how clinicians manage ITP in a real-world setting without the constraints of a clinical trial protocol. References 1. Boccia R, et al. Br J Haematol. 2020. 2. Bussel J, et al. Am J Hematol. 2019 Figure 1 Figure 1. Disclosures Hughes: Abbvie: Speakers Bureau; Rigel: Other: Advisory Board, Research Funding; Amgen: Speakers Bureau; Karyopharm: Other: Advisory Board, Speakers Bureau. Numerof: Rigel Pharmaceuticals, Inc.: Current Employment, Current equity holder in publicly-traded company. Zider: Rigel Pharmaceuticals, Inc.: Current Employment, Current equity holder in publicly-traded company. Chow: Rigel Pharmaceuticals, Inc.: Current Employment, Current equity holder in publicly-traded company. Goel: Rigel Pharmaceuticals, Inc.: Consultancy; Alexion: Consultancy; NHLBI: Research Funding.

scholarly journals Dasatinib in First- and Second-Line Therapy of Chronic Myeloid Leukemia: Efficacy, Safety and Quality of Life

2017 ◽  
Vol 10 (2) ◽  
pp. 206-217
Author(s):  
TI Ionova ◽  
◽  
NB Bulieva ◽  
OYu Vinogradova ◽  
TA Gritsenko ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Chronic Myeloid Leukemia ◽  
Myeloid Leukemia ◽  
Second Line ◽  
Second Line Therapy ◽  
Line Therapy

40-OR: Quality of Life in People with Type 2 Diabetes following Initiation of Second-Line Therapy: DISCOVER

Diabetes ◽  
2020 ◽  
Vol 69 (Supplement 1) ◽  
pp. 40-OR
Author(s):  
ANTONIO NICOLUCCI ◽  
HUNGTA CHEN ◽  
ANDREW COOPER ◽  
MARILIA B. GOMES ◽  
LINONG JI ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Type 2 Diabetes ◽  
Second Line ◽  
Second Line Therapy ◽  
Line Therapy

scholarly journals Anxiety in Adult Patients Living with ITP Stratified across Different Treatment Types and Groups

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 18-18
Author(s):  
Jennifer Diraimo ◽  
Caroline Kruse ◽  
Michele P. Lambert ◽  
Alexandra Kruse
Keyword(s):  
Quality Of Life ◽  
Board Of Directors ◽  
Research Funding ◽  
Second Line ◽  
First Line ◽  
Second Line Therapy ◽  
Advisory Committees ◽  
Treatment History ◽  
Line Therapy

Background:Immune thrombocytopenia purpura (ITP) is an autoimmune bleeding disorder that is heterogeneous in presentation, disease course, treatment response, and impact on quality of life. Treatments often cause unpleasant side effects, and prolonged use can lead to tolerability issues and toxicity. Disease symptoms and treatments available impact the health-related quality of life (HRQoL) among patients living with ITP. Here, we compare anxiety and its impact among adult ITP patients and determine whether anxiety levels differ dependent on treatment. Methods: For this study, we used three of the five surveys from the PDSA Natural History Study Registry including; treatment history and two adult QoL surveys. As of June 2020, 357 adult patients completed the survey assessing treatment history. A total of 310 adult patients completed the adult QoL (part one) survey, and 301 adults completed the adult QoL (part two) survey. Patients were stratified by 1) no treatment received 2) treated in the past 3) on therapy within the last six months; patients currently on therapy were further stratified by first and second-line therapy. Results: Among the 357 completed surveys on treatment history, 11% (n=40) have never received treatment for ITP, 46% (n=166) have in past, and 43% (n=158) currently receive therapy (within the last six months). Among those currently on treatment, 82% receive monotherapy; 26% use a first-line therapy (corticosteroid, IVIG, or Anti-D), and 43% are using a second-line therapy (TPO-RA, rituximab, and other second-line options). Therapies reported include TPO-RA's (41%), corticosteroids (24%), IVIG (7%), rituximab (3%), SYK inhibitor (1%), antibiotics (4%), anti-D (1%) and other second-line treatments (such as MMF), and "other" treatments including complementary therapies (14%). Overall, 23% had a splenectomy at some point to manage their active ITP. When asked to reflect on the last seven days, patients completing the QoL survey (n=310), 66% felt anxious; 17% reported this was experienced 'almost always/often'. Among those who have never been treated, feeling anxious was reported 67% of the time; 18% reported feeling this way 'almost always/often'. A similar trend was observed in patients not currently on treatment. Among those receiving a first line therapy, anxiousness was reported 74% overall; 19% 'almost always/often'. Among those receiving a second line therapy, 72% reported feeling anxious; 9% reported feeling this way 'almost always/often'. Differences in high levels of anxiousness reported among the different treatment groups was not significant (X2= 3.4, p=.48). Difficulties focusing were reported (51%, 9% reporting this occurred 'almost always/often'). Among those who have never been treated, difficulties were reported (48%, 12%, 'almost always/often'). Those not currently receiving treatment had difficulties focusing due to anxiety (50%, 4% reporting this 'almost always/often'). Those on first line treatment indicated focus was impacted by anxiety overall (60%, 36% 'almost always/often') and those receiving second-line therapy reported (58%, 8% 'almost always/often'). Differences in high levels of anxiety affecting concentration reported among the treatment groups was significant (X2= 20.87, p=.00033), revealing a higher anxiety profile among those using corticosteroids. When difficulty with focus due to anxiety was compared between those receiving corticosteroids and those receiving a TPO-RA specifically, anxiety was significantly higher in the steroid group (X2=9.15, P=.0024); this trend was not found to be statistically significant among other second line therapies. Conclusion: The physical symptoms of ITP often guide treatment selection for patients however, providers should also focus on mitigating stress and other indicators of mental health in order to provide the best outcome and quality of life in disease course. Differences in interpretation behind the terms feeling 'anxious' vs ' anxiety affecting focus' may explain our conflicting results. Higher anxiety levels (in contrast to higher anxiousness) appeared related to treatment type in those currently receiving therapy; corticosteroid users were more impacted by their anxiety than those receiving TPO-RAs; steroids are known to interfere with mood and concentration, and this is confirmed by patients in this survey. Disclosures Kruse: CSL Behring: Other: Grant paid to PDSA; UCB: Other: Grant and consultancy fee, all paid to PDSA; Rigel: Other: Grant paid to PDSA; Principia: Other: Grant paid to PDSA; Pfizer: Other: Grant and consultancy fee, all paid to PDSA; Argenx: Other: Grant paid to PDSA; Amgen: Other: Grant and honorarium, all paid to PDSA; Novartis: Other: PDSA received payment for recruiting patients to I-WISh and for promoting I-WISh on the globalitp.org website. Grant and consultancy fee, all paid to PDSA outside the submitted work. Lambert:AstraZeneca: Research Funding; Principia: Consultancy, Membership on an entity's Board of Directors or advisory committees; Dova: Consultancy, Membership on an entity's Board of Directors or advisory committees; Shionogi: Consultancy; Sysmex: Research Funding; RDMD ITP study: Consultancy; ITP Australia: Consultancy; CdLS Foundation: Consultancy; 22qSociety: Consultancy; Platelet Disorder Support Association (PDSA): Consultancy; ClinGen: Honoraria; Bayer: Consultancy; Argenix: Consultancy; Octapharma: Consultancy, Research Funding; Educational Concepts in Medicine: Consultancy; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding.

scholarly journals Incidence of Hypoglycemia and Hospitalization Related to Chronic Diabetes Complications and Its Effect on Quality of Life Among Patients Initiating Second Line Therapy: DISCOVER Study

2021 ◽  
Author(s):  
Khalid Al-Rubeaan ◽  
Faisal Banah ◽  
Fayez Alruwaily ◽  
Eman Sheshah ◽  
Dhekra Alnaqeb ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Type 2 Diabetes ◽  
Metabolic Control ◽  
Annual Incidence ◽  
Second Line ◽  
Second Line Therapy ◽  
Fear Of Hypoglycemia ◽  
Line Therapy

Abstract The management of patients with type 2 diabetes is a complex process that must be individualized and be patient centered. The aim of this study was to assess the metabolic control, the annual incidence and crude prevalence of hypoglycemia, hospitalization, and complications among patients with type 2 diabetes initiating second-line therapy. This study is an observational, longitudinal, prospective study as a part of the multinational DISCOVERing Treatment Reality of Type 2 Diabetes in Real World Settings (DISCOVER) study, that recruited 519 patients with type 2 diabetes who were non-insulin users, aged ≥18 years, and switching to second-line therapy. The cohort was clinically evaluated over three years of follow up. Fear of hypoglycemia was assessed using the Hypoglycemia Fear Survey (HFS II), while the quality of life was assessed using SF36v2 questionnaire. Using second-line therapy improved metabolic control but the annual incidence of microangiopathies were at 61/1000 patient-years, 47/1000 patient-years, and 4/1000 patient-years for neuropathy, retinopathy, and nephropathy, respectively. The incidence of hypoglycemia was 57/1000 patient-years, where 50% were recurrent during the three-years period. The HFS II showed a significant increase in patients’ worries related to hypoglycemia. The incidence of hospitalization was 31/1000 patient-years, out of which 8/1000 patient-years were related to cardiovascular events, mainly myocardial infarction. Moderate metabolic control was associated with lower incidence of macro angiopathy and an increased incidence and fear of hypoglycemia, while it was associated with improved mental component score when assessing the patients’ quality of life. The treating physician’s decision of treatment intensification should be individualized with consideration of befits of good glycemic control versus the risk of hypoglycemia, especially in elderly patients.

Bendamustine Induces Higher Remission Rates, Prolongs Progression Free Survival as Well as Time to Next Treatment, and Improves Overall Survival for Patients In Complete Remission without Compromising Quality of Life When Compared to Chlorambucil In First Line Treatment of Chronic Lymphocytic Leukemia

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2449-2449 ◽  
Author(s):  
Wolfgang Ulrich Knauf ◽  
Toshko Lissitchkov ◽  
Ali Aldaoud ◽  
Anna Marina Liberati ◽  
Javier Loscertales ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Progression Free Survival ◽  
Treated Group ◽  
Lymphocytic Leukemia ◽  
Second Line ◽  
First Line ◽  
Second Line Therapy ◽  
Free Survival ◽  
Line Therapy

Abstract Abstract 2449 Introduction: Bendamustine (BEN), either alone or in combination with Rituximab, is increasingly used in the treatment of chronic lymphocytic leukemia (CLL) and various types of low grade Non Hodgkin's Lymphoma (NHL). The approval to treat CLL with BEN is based on a prospectively randomized trial (Knauf et al., J Clin Oncol. 2009; 27: 4378–4384) comparing single drug BEN with chlorambucil (CLB). Here, we report on follow-up data of this pivotal trial with specific reference to survival times, time to next treatment, and efficacy of second line regimens. Since CLL is a disease of the elderly and potentially co-morbid patients, we also analyzed quality of life (QoL) parameters in relation to the treatment with both BEN and CLB. Patients and Methods: The efficacy and safety of BEN and CLB have been compared in a randomized, open-label, multicenter, phase III trial in patients with previously untreated advanced (Binet stage B/C) CLL. Patients were randomized to receive either BEN (100 mg/m2 on days 1 + 2) or CLB (0.8 mg/kg on days 1 and 15) for up to 6 treatment cycles. The primary endpoints were overall response rate (ORR), which was defined as complete (CR) or partial response (PR), and progression-free survival (PFS). Secondary endpoints included overall survival (OS), and QoL. The latter was analyzed by using both the EORTC questionnaires QLQ C30 and QLQ-CLL25. We also looked at time to next treatment and efficacy of second line regimens. The response to treatment was evaluated by a blinded Independent Response Assessment Committee. Results: A total of 319 patients were randomized (162 BEN and 157 CLB), all of whom were included in the efficacy analysis, while 308 patients were evaluable for QoL analysis (158 BEN and 150 CLB). Median age was 64 years (range 35 to 78). The mean number of treatment cycles was 5 in both study arms, regardless of an age above or below 65 years. The median observation time was 54 months. ORR was significantly higher with BEN than with CLB (68% versus 31%, P<0.0001). A CR was achieved in 31% of pts with BEN and in 2% of pts with CLB (P<0.0001). In the intent to treat (ITT) population, the median PFS was 21.2 months with BEN and 8.8 months with CLB (P<0.0001). Sixty-three patients in the BEN treated group and 35 in the CLB treated group had not received any second line therapy (P<0.001) at the time of this analysis. The median time to next treatment in the ITT population was 31.5 months with BEN and 10.1 months with CLB (P<0.0001). ORR after second line therapy of any type was 35.4% in the BEN first line arm and 45.9% in the CLB first line arm (P=0.131). So far, there is no difference in OS (P = 0.24; hazard ratio = 1.3 in favour of BEN) in the ITT population. However, patients achieving a CR (almost exclusively after BEN) experienced a longer OS than pts not in CR (median not reached versus 76.2 months; P=0.002). Also, pts with any response (CR + PR) either after BEN or CLB had a longer OS than the non-responders (median not reached versus 68.3 months; P< 0.0001). Base line scores regarding QoL parameters showed no difference between the groups. After completion of study treatment (mean 5 cycles administered), no differences became evident with respect to physical, social, emotional, and cognitive functioning. The self assessment of the global health status also revealed no difference. Conclusion: This study has shown that BEN offers significantly greater response rates, PFS, and a much longer time to next treatment than CLB. OS is prolonged significantly in all responders and especially in those patients who achieve CR after BEN. In comparison to CLB, the additional efficacy of BEN was achieved without compromising QoL. BEN should be considered as a backbone drug in first-line chemotherapy of patients with advanced CLL. Disclosures: Knauf: Mundipharma: Consultancy, Honoraria. Klein: Mundipharma: Honoraria. Merkle: Mundipharma: Honoraria.

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