Patient-Reported Outcomes of Deferasirox (Exjade®, ICL670) versus Deferoxamine in Sickle Cell Disease Patients with Transfusional Hemosiderosis

2008 ◽  
Vol 119 (3) ◽  
pp. 133-141 ◽  
Author(s):  
Elliott Vichinsky ◽  
Zahra Pakbaz ◽  
Onyinye Onyekwere ◽  
John Porter ◽  
Paul Swerdlow ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Cell Disease ◽  
Patient Reported

scholarly journals End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain

2019 ◽  
Vol 3 (23) ◽  
pp. 3982-4001 ◽  
Author(s):  
Ann T. Farrell ◽  
Julie Panepinto ◽  
C. Patrick Carroll ◽  
Deepika S. Darbari ◽  
Ankit A. Desai ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Cell Disease ◽  
End Points ◽  
Additional Clinical Trial ◽  
Patient Reported ◽  
The Brain

Abstract To address the global burden of sickle cell disease (SCD) and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to: patient-reported outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.

POST Marketing Observational Study of Children (6 years or older) Treated with Deferasirox (Exjade ICL 670)-One Year analysis

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 5295-5295
Author(s):  
Nagina Parmar ◽  
Manroop Singh ◽  
Isaac Odame ◽  
Melanie Kirby
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Chelation Therapy ◽  
Descriptive Statistics ◽  
Cell Disease ◽  
Outcomes Measures ◽  
Short Period ◽  
Patient Reported

Abstract Abstract 5295 In 2006, deferasirox was licensed in Canada for use in children over the age of 6 years. Deferasirox (Exjade â, ICL 670) is an orally active iron chelators which represents the new class of tridentate iron chelator. Deferasirox has been formulated as a dispersible tablet based on technical feasibility, stability, and adequate bioavailability. Deferasirox has been shown to be as effective as deferral (DFO) and has a clinically manageable safety profile (the most common side effects are gastrointestinal disturbances and rashes). Few studies have been conducted to compare the satisfaction, convenience, activity limitations, and patient preferences of deferasirox in comparison with DFO in sickle cell and thalassemia patients. The purpose of the present study was to evaluate the long-term response of deferosirox in iron-overloaded children aged 6 years or older treated with transfusion dependent anemia and secondary objective was to collect and analyze the following parameters such as serum ferrtin, SLT, AST, creatinine, audiology and ophthalmology, liver iron concentrations (LIC) and patient reported outcomes measures over a period of 2 years from the start of deferasirox. The study population comprised of male and female patients aged 6 years or <17.99 with chronic iron overload related to blood transfusions in patients with Thalassemia and Sickle cell disease. Fifty-one patients were enrolled into this study at the Hematology Clinic, Hospital for Sick Children, Toronto, ON Canada. This study was observational and does not impose a therapy protocol, diagnostic/therapeutic interventions or a strict visit schedule. Patients were treated with deferasirox according to the local physician's judgment and in accordance with the local (country-specific) deferasirox prescribing information. Data were collected at study entry (baseline), and at 12 months. Patients were approached for the study after the ethics approval was obtained. All variables were explored and summarized using descriptive statistics such as means, standard deviation, median and ranges, counts and proportions, graphs as appropriate. Patients reported outcomes measures questionnaires were given after the written consent/assent was obtained. Statistical analysis was done by descriptive statistics and paired t-test. Of the 51 subjects, 30 were females, 21 were males, and median age for the patients was 14 ± 5.3. Out of 51 patients, 49 patients had thalassemia and two had sickle cell disease. All of the subjects were on desferal chelation therapy before the start of the deferasirox, Analysis of patient reported outcomes measures showed that majority of the patients (73%) were very satisfied with deferasirox, when they started and after 12 months on the study, however 1 patient who reported dissatisfaction in 2009, reported satisfaction in year 2010. The main reason for the deferasirox, treatment preference were relief of pain associated with injections (30%) and more convenient (35%) in administration. Other reasons given were improved sleep patterns (2%) and less disruption to the family (6%). At baseline and 12 months laboratory evaluations were as follows: Mean Creatinine values (88% of the patients) (baseline- 34.41±11.49, 12 months – 52.41 ± 19.82), Mean ALT (65% of the patients) (baseline- 41.73 ± 7.32, 12 months – 30.94 ± 5.30) and serum ferritin (baseline- 1995± 276, 12 month, 1833 ± 203). Mean LIC (baseline-11.08 ± 1.29, 12 months- 7.87 ± 1.12) evaluations showed the incremental decrease over time with 14/51 showed the values of <3 over a 12 months period. Chelation therapy was held for some patients for a short period to prevent toxicity related to the chelator. Mild (4/51) and moderate (1/51) hearing loss were observed in subjects over period from 2009–2010. Data will be collected after 24 months to evaluate their long-term responses and for comparative analysis. Further, we can conclude that given the high levels of satisfaction, it is likely that the quality of life and adherence to chelation treatment have improved for patients who are receiving the deferasirox treatment compared with previous subcutaneous chelation treatment. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 196-205
Author(s):  
Sherif M. Badawy
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stakeholder Engagement ◽  
Patient Reported Outcomes ◽  
Outcomes Assessment ◽  
Cell Disease ◽  
Patient Centered ◽  
Patient Reported

Abstract Patients with sickle cell disease (SCD) have significant impairment in their quality of life across the life span as a consequence of serious disease burden with several SCD-related complications. A number of disease-modifying therapies are currently available, yet long-term clinical benefits in real-world settings remain unclear. Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including: (1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (consensus measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient-reported outcomes assessment is highly recommended, and patient-reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations for Interventional Trials guidelines. Patient-centered outcomes research (PCOR) approaches and meaningful stakeholder engagement throughout the process have the potential to optimize the execution and success of clinical trials in SCD with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient-centered research approaches and stakeholder engagement. A proposed hematology stakeholder-engagement framework for clinical trials is also discussed.

scholarly journals Evaluation of Longitudinal Pain Study in Sickle Cell Disease (ELIPSIS) by Electronic Patient-Reported Outcomes, Actigraphy, and Biomarkers

Blood ◽  
2020 ◽  
Author(s):  
Debra Pittman ◽  
Patrick C Hines ◽  
David Roger Beidler ◽  
Denis Rybin ◽  
Andrew L Frelinger ◽  
...  
Keyword(s):  
Health Care ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Health Care Utilization ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Care Utilization ◽  
Cell Disease ◽  
Patient Reported ◽  
At Home

Clinical trials in sickle cell disease (SCD) often focus on health care utilization for painful vaso-occlusive crises (VOC). However, no objective, quantifiable pain biomarkers exist, pain is not specific to VOCs, health care utilization varies between patients, unreported at-home VOCs likely contribute to long-term outcomes, and patient-reported outcomes are seldom considered. This non-interventional, longitudinal, 6-month study aimed to develop tools to identify VOCs in SCD patients with or without health care utilization. Participants wore an actigraph device, tracking sleep and activity. SCD patients used an electronic patient-reported outcome (ePRO) tool collecting pain, medication, fatigue, and daily function. Patients self-reported when they experienced VOC pain (VOC day). Biomarkers were collected every 3 weeks (non-VOC). Self-reported VOCs triggered at-home or in-hospital blood collection. The study enrolled 37 participants with SCD; 35 completed the study. Participants reported 114 VOC events and 346 VOC days, of which 62.3% and 78.3%, respectively, were self-treated at home. The ePRO and actigraphy captured endpoints of pain, functionality, fatigue, activity, and sleep; each was significantly altered on VOC days compared with non-VOC days. Biomarkers collected at home or in hospital on VOC days were significantly altered compared with non-VOC baseline values, including leukocyte-platelet aggregates, microfluidic-based blood cell adhesion, interleukin-6, C-reactive protein, interleukin-10, tumor necrosis factor-alpha, and thrombin-antithrombin. ELIPSIS: demonstrates the feasibility of accurately monitoring out-of-hospital pain, using patient-reported VOC days as potential endpoints for clinical trials in SCD; showed changes in biomarkers and activity measured by actigraphy that may enable improved identification and assessment of VOCs.

scholarly journals Challenges to management of pain in sickle cell disease

2018 ◽  
Vol 8 (1) ◽  
Author(s):  
Julie Kanter
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Medical Providers ◽  
Cell Disease ◽  
Blood Disorders ◽  
Pain Experience ◽  
Multiple Dimensions ◽  
Patient Reported ◽  
Pharmacologic Agents

Sickle Cell Disease (SCD) is one of the most common blood disorders in the world. Pain is the primary reason for which individuals with SCD interact with the healthcare system. Generally speaking, there are two types of SCD pain: vaso-occlusive pain (or sickle cell disease crisis) and chronic pain caused by an accumulation of organ and tissue damage over time. However, despite its frequency, we have limited understanding about what causes pain in sickle cell disease, how best to manage pain in SCD and (most importantly) how to prevent pain in SCD. For medical providers, pain is also an elusive target due to the difficulty in objectively measuring pain and the importance of relying on patient reported outcomes. To face the challenges in managing pain in SCD, we will review the current understanding of the pathophysiology of vaso-occlusion, the multiple dimensions of the pain experience, and the current methods of measuring and managing pain. We will also review new pharmacologic agents undergoing clinical trials in SCD that will help to prevent pain and improve outcomes in SCD.

scholarly journals What is the future of patient-reported outcomes in sickle-cell disease?

2020 ◽  
Vol 13 (11) ◽  
pp. 1165-1173
Author(s):  
Sharon A. Singh ◽  
Nitya Bakshi ◽  
Prashant Mahajan ◽  
Claudia R. Morris
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Cell Disease ◽  
Patient Reported ◽  
The Future
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