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Changing Paradigms in ITP Management: Newer Tools for an Old Disease.

ABSTRACT: Immune thrombocytopenia (ITP) is an autoimmune disease characterized by isolated thrombocytopenia that may be accompanied clinically by bleeding and reduced health-related quality of life (HRQoL). While corticosteroids, splenectomy, and various immunosuppressants (used off-label) have served as historical mainstays of ITP treatment, their use is associated with adverse effects and morbidity. Over the last 15 years, the advent of the thrombopoietin receptor agonists has revolutionized the management of chronic ITP with high response rates, durable responses, and minimal adverse effects in most patients. With four agents now FDA-approved to manage chronic ITP, there is a renewed emphasis on improving HRQoL and minimizing the toxicities associated with traditional therapies. Promising agents with diverse mechanisms of action, ranging from those targeting Bruton's Tyrosine Kinase to the neonatal Fc receptor, are currently under investigation. This review highlights recent landmark clinical trials which have made significant impacts on ITP management and ongoing drug development. In critically analyzing studies of relevance, we illustrate the changing paradigms of ITP management and how the field is advancing beyond traditional therapies.

SUBMITTER: Jiang D

PROVIDER: S-EPMC10044485 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature

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Changing Paradigms in ITP Management: Newer Tools for an Old Disease.

Jiang Debbie D Al-Samkari Hanny H Panch Sandhya R SR

Transfusion medicine reviews 20220914 4

Immune thrombocytopenia (ITP) is an autoimmune disease characterized by isolated thrombocytopenia that may be accompanied clinically by bleeding and reduced health-related quality of life (HRQoL). While corticosteroids, splenectomy, and various immunosuppressants (used off-label) have served as historical mainstays of ITP treatment, their use is associated with adverse effects and morbidity. Over the last 15 years, the advent of the thrombopoietin receptor agonists has revolutionized the managem ...[more]

PMID: 36273934

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Project description:Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia caused by increased platelet destruction and impaired platelet production. First-line therapies include corticosteroids, intravenous immunoglobulin, and anti-D immunoglobulin. For patients who are refractory to these therapies, those who become corticosteroid dependent, or relapse following treatment with corticosteroid, options include splenectomy, rituximab, and thrombopoietin-receptor agonists, alongside a variety of additional immunosuppressive and experimental therapies. Despite recent advances in the management of ITP, many areas need further research. Although it is recognized that an assessment of patient-reported outcomes in ITP is valuable to understand and guide treatment, these measures are not routinely measured in the clinical setting. Consequently, although corticosteroids are first-line therapies for both children and adults, there are no data to suggest that corticosteroids improve health-related quality of life or other patient-related outcomes in either children or adults. In fact, long courses of corticosteroids, in either children or adults, may have a negative impact on a patient's health-related quality of life, secondary to the impact on sleep disturbance, weight gain, and mental health. In adults, additional therapies may be needed to treat overt hemorrhage, but unfortunately the results are transient for the majority of patients. Therefore, there is a need to recognize the limitations of current existing therapies and evaluate new approaches, such as individualized treatment based on the probability of response and the size of effect on the patient's most bothersome symptoms and risk of adverse effects or complications. Finally, a validated screening tool that identifies clinically significant patient-reported outcomes in routine clinical practice would help both patients and physicians to effectively follow a patient's health beyond simply treating the laboratory findings and physical symptoms of ITP. The goal of this narrative review is to discuss management of newly diagnosed and refractory patients with ITP, with a focus on the limitations of current therapies from the patient's perspective.

Project description:Balancing economic, ecological, and social values has long been a challenge in the forests of the Pacific Northwest, where conflict over timber harvest and old-growth habitat on public lands has been contentious for the past several decades. The Northwest Forest Plan, adopted two decades ago to guide management on federal lands, is currently being revised as the region searches for a balance between sustainable timber yields and habitat for sensitive species. In addition, climate change imposes a high degree of uncertainty on future forest productivity, sustainability of timber harvest, wildfire risk, and species habitat. We evaluated the long-term, landscape-scale trade-offs among carbon (C) storage, timber yield, and old forest habitat given projected climate change and shifts in forest management policy across 2.1 million hectares of forests in the Oregon Coast Range. Projections highlight the divergence between private and public lands under business-as-usual forest management, where private industrial forests are heavily harvested and many public (especially federal) lands increase C and old forest over time but provide little timber. Three alternative management scenarios altering the amount and type of timber harvest show widely varying levels of ecosystem C and old-forest habitat. On federal lands, ecological forestry practices also allowed a simultaneous increase in old forest and natural early-seral habitat. The ecosystem C implications of shifts away from current practices were large, with current practices retaining up to 105 Tg more C than the alternative scenarios by the end of the century. Our results suggest climate change is likely to increase forest productivity by 30-41% and total ecosystem C storage by 11-15% over the next century as warmer winter temperatures allow greater forest productivity in cooler months. These gains in C storage are unlikely to be offset by wildfire under climate change, due to the legacy of management and effective fire suppression. Our scenarios of future conditions can inform policy makers, land managers, and the public about the potential effects of land management alternatives, climate change, and the trade-offs that are inherent to management and policy in the region.

Dataset Information

Changing Paradigms in ITP Management: Newer Tools for an Old Disease.

Publications

Changing Paradigms in ITP Management: Newer Tools for an Old Disease.

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