Comparative effectiveness research in the United States and primary immunodeficiency diseases

2011 ◽  
Vol 13 (3,4) ◽  
pp. 183-191
Author(s):  
Emily Hovermale ◽  
Christopher Scalchunes ◽  
Marcia Boyle
Keyword(s):  
United States ◽  
Primary Immunodeficiency ◽  
Comparative Effectiveness Research ◽  
Comparative Effectiveness ◽  
The United States ◽  
Effectiveness Research ◽  
Primary Immunodeficiency Diseases ◽  
Immunodeficiency Diseases

scholarly journals Neutropenia Is an Under-Recognized Finding in Pediatric Primary Immunodeficiency Diseases: An Analysis of the United States Immunodeficiency Network Registry

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3685-3685
Author(s):  
Thomas F. Michniacki ◽  
Kelly J. Walkovich ◽  
Julie Sturza ◽  
Lauren E. Merz ◽  
James Connelly ◽  
...  
Keyword(s):  
United States ◽  
Innate Immunity ◽  
Primary Immunodeficiency ◽  
Pediatric Patients ◽  
The United States ◽  
Common Element ◽  
Severe Neutropenia ◽  
Primary Immunodeficiency Diseases ◽  
Immunodeficiency Diseases ◽  
Significant Difference

Abstract Background: Neutropenia is recognized as a defining or common element in a limited number of primary immunodeficiency diseases (PIDDs) (Dotta L et al. Immunol Let 2014; Cham B et al. Semin Hematol 2002); but, the overall prevalence of neutropenia within the more than 300 known PIDDs is not well described. Many of the PIDDs rely on a high index of clinical suspicion for diagnosis which may not be readily apparent to hematologists evaluating a neutropenic patient, underscoring the importance of understanding the rate of neutropenia in PIDDs and impact on clinical outcomes. The goal of this study was to evaluate the frequency and characteristics of neutropenia in a large registry of patients with primary immunodeficiency (PID). Methods: The United States Immunodeficiency Network (USIDNET) is a research consortium that maintains a national registry for the collection of data on patients with PIDDs. The USIDNET was queried for data regarding patients aged 21 years or less. Data utilized included reported PID diagnosis, gene mutation, gender, race, status of patient (living or deceased), year of birth, age at death, age of disease diagnosis, and results of reported complete blood counts (CBCs). PID diagnoses were grouped according to the 2017 International Union of Immunological Societies (IUIS) Phenotypic Classification for Primary Immunodeficiencies (Bousfiha A et al. J Clin Immunol 2018). Mild, moderate, and severe neutropenia were defined as absolute neutrophil counts of 1000-1499 cells/μL, 500-999 cells/μL, and < 500 cells/μL, respectively. Data are presented with descriptive statistics. The frequency of neutropenia was compared between genders and by race using chi-square test analysis. Results: An initial data request resulted in information on 1577 patients; 384 patients were eliminated from analysis given insufficient recorded registry data due to a lack of a documented absolute neutrophil count. An additional 48 patients were excluded given that complete blood cell counts were only reported following hematopoietic stem cell transplantation. Data was thus available for 1145 pediatric patients with PIDDs. A total of 2058 neutrophil values were analyzed with 62.7% (719) patients having only a single neutrophil value available for review. Overall, 15.8% of PIDD pediatric patients in the USIDNET registry had neutropenia, ranging from 12.01% (combined immunodeficiencies with associated or syndromic features) to 66.67% (defects in intrinsic and innate immunity) in IUIS categories (Table 1) and in USIDNET categories from 2.94% (autoimmune lymphoproliferative syndrome) to 75.00% (susceptibility to mycobacterial infections) (Table 2). Of PID patients with neutropenia, 20.4% had severe neutropenia, 33.1% had moderate neutropenia, and 46.4% had mild neutropenia. Neutropenia was most likely to be severe in patients with predisposition to severe viral infections as well as those with a defect in intrinsic and innate immunity (Tables 1-2). There was no statistically significant difference in neutropenia between genders (p = 0.64). However, females were more likely to have severe neutropenia than males (28% versus 16%; p=0.03). Additionally, 28% of African-American patients had recorded neutropenia while only 17% of Caucasians and 2% of Asian/Pacific Islanders were neutropenic (p=0.005). There was no statistically significant difference in severity of neutropenia and death nor in the rate of bacterial and fungal infections detected between neutropenic and non-neutropenic patients with 93% of patients in the dataset living at the time of data collection. Conclusions: The frequency of neutropenia within pediatric patients diagnosed with PIDD is higher and more ubiquitous than previously suspected. Hematologists should consider a PIDD in the assessment of neutropenia, particularly if other concerning signs or symptoms are present. Although this evaluation did not detect a difference in morbidity or mortality, the data set was constrained by a limited number of CBCs per patient and the rarity of the PIDDs. The impact of neutropenia on PIDD outcomes requires further study, ideally through a prospective longitudinal study. Acknowledgments: The U.S. Immunodeficiency Network (USIDNET), a program of the Immune Deficiency Foundation (IDF), is supported by a cooperative agreement, U24AI86837, from the National Institute of Allergy and Infectious Diseases (NIAID). Disclosures Dale: Athelas, Inc.: Equity Ownership; Amgen: Consultancy, Research Funding; Sanofi-Aventi: Consultancy, Honoraria; Cellerant: Other: Scientific Advisory Board; Hospira: Consultancy; Prolong: Consultancy; Beheringer-Ingelheim: Consultancy; Coherus: Consultancy.

scholarly journals Facts, Fallacies, and Politics of Comparative Effectiveness Research: Part 2 - Implications for Interventional Pain Management

2010 ◽  
Vol 1;13 (1;1) ◽  
pp. E55-E79
Author(s):  
Laxmaiah Manchikanti
Keyword(s):  
United States ◽  
Health Care ◽  
Pain Management ◽  
Comparative Effectiveness Research ◽  
Comparative Effectiveness ◽  
Clinical Excellence ◽  
The United States ◽  
Effectiveness Research ◽  
Interventional Pain Management ◽  
Care Costs

The United States leads the world in many measures of health care innovation. However, it has been criticized to lag behind many developed nations in important health outcomes including mortality rates and higher health care costs. The surveys have shown the United States to outspend all other Organisation for Economic Co-operation and Development (OECD) countries with spending on health goods and services per person of $7,290 – almost 2½ times the average of all OECD countries in 2007. Rising health care costs in the United States have been estimated to increase to 19.1% of gross domestic product (GDP) or $4.4 trillion by 2018. CER is defined as the generation and synthesis of evidence that compares the benefits and harms of alternate methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The, comparative effectiveness research (CER) has been touted by supporters with high expectations to resolve most ill effects of health care in the United States providing high quality, less expensive, universal health care. The efforts of CER in the United States date back to the late 1970s and it was officially inaugurated with the enactment of the Medicare Modernization Act (MMA). It has been rejuvenated with the American Recovery and Reinvestment Act (ARRA) of 2009 with an allocation of $1.1 billion. CER has been the basis of decision for health care in many other countries. Of all the available agencies, the National Institute for Health and Clinical Excellence (NICE) of the United Kingdom is the most advanced, stable, and has provided significant evidence, though based on rigid and proscriptive economic and clinical formulas. While CER is taking a rapid surge in the United States, supporters and opponents are emerging expressing their views. Since interventional pain management is a new and evolving specialty, with ownership claimed by numerous organizations, at times it is felt as if it has many fathers and other times it becomes an orphan. Part 2 of this comprehensive review will provide facts, fallacies, and politics of CER along with discussion of potential outcomes, impact of CER on health care delivery, and implications for interventional pain management in the United States. Key words: Comparative effectiveness research, evidence-based medicine, Institute of Medicine, National Institute for Health and Clinical Excellence, interventional pain management, interventional techniques, geographic variations, inappropriate care.

scholarly journals Facts, Fallacies, and Politics of Comparative Effectiveness Research: Part I. Basic Considerations

2010 ◽  
Vol 1;13 (1;1) ◽  
pp. E23-E54
Author(s):  
Laxmaiah Manchikanti
Keyword(s):  
United States ◽  
Health Care ◽  
Medical Care ◽  
Comparative Effectiveness Research ◽  
Comparative Effectiveness ◽  
Clinical Excellence ◽  
The United States ◽  
Evidence Based ◽  
Effectiveness Research ◽  
Care Costs

While the United States leads the world in many measures of health care innovation, it has been suggested that it lags behind many developed nations in a variety of health outcomes. It has also been stated that the United States continues to outspend all other Organisation for Economic Co-operation and Development (OECD) countries by a wide margin. Spending on health goods and services per person in the United States, in 2007, increased to $7,290 – almost 2½ times the average of all OECD countries. Rising health care costs in the United States have been estimated to increase to 19.1% of gross domestic product (GDP) or $4.4 trillion by 2018. The increases are illustrated in both public and private sectors. Higher health care costs in the United States are implied from the variations in the medical care from area to area around the country, with almost 50% of medical care being not evidence-based, and finally as much as 30% of spending reflecting medical care of uncertain or questionable value. Thus, comparative effectiveness research (CER) has been touted by supporters with high expectations to resolve most ill effects of health care in the United States and provide high quality, less expensive, universal health care. CER is defined as the generation and synthesis of evidence that compares the benefits and harms of alternate methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The efforts of CER in the United States date back to the late 1970’s even though it was officially born with the Medicare Modernization Act (MMA) and has been rejuvenated with the American Recovery and Reinvestment Act (ARRA) of 2009 with an allocation of $1.1 billion. CER has been the basis for health care decision-making in many other countries. According to the International Network of Agencies for Health Technology Assessments (INAHTA), many industrialized countries have bodies that are charged with health technology assessments (HTAs) or comparative effectiveness studies. Of all the available agencies, the National Institute for Health and Clinical Excellence (NICE) of the United Kingdom is the most advanced, stable, and has provided significant evidence, though based on rigid and proscriptive economic and clinical formulas. While CER is making a rapid surge in the United States, supporters and opponents are expressing their views. Part I of this comprehensive review will describe facts, fallacies, and politics of CER with discussions to understand basic concepts of CER. Key words: Comparative effectiveness research, evidence-based medicine, Institute of Medicine, National Institute for Health and Clinical Excellence, interventional pain management, interventional techniques, geographic variations, inappropriate care.

scholarly journals The Impact of Comparative Effectiveness Research on Interventional Pain Management: Evolution from Medicare Modernization Act to Patient Protection and Affordable Care Act and the Patient-Centered Outcomes Research Institute

2011 ◽  
Vol 3;14 (3;5) ◽  
pp. E249-E282
Author(s):  
Laxmaiah Manchikanti
Keyword(s):  
United States ◽  
Pain Management ◽  
Affordable Care Act ◽  
Comparative Effectiveness Research ◽  
Outcomes Research ◽  
Comparative Effectiveness ◽  
The United States ◽  
Effectiveness Research ◽  
Patient Centered ◽  
Interventional Pain Management

The Patient-Centered Outcomes Research Institute (PCORI) was established by the Affordable Care Act of 2010 to promote comparative effectiveness research (CER) to assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis. The development of PCORI is vested in the Medicare Modernization Act (MMA) and the American Recovery and Reinvestment Act (ARRA). The framework of CER and PCORI describes multiple elements which are vested in all 3 regulations including stakeholder involvement, public participation, and open transparent decision-making process. Overall, PCORI is much more elaborate with significant involvement of stakeholders, transparency, public participation, and open decision-making. However, there are multiple issues concerning the operation of such agencies in the United States including the predecessor of Agency for Healthcare Research and Quality (AHRQ), the Agency for Healthcare Policy and Research (AHCPR), AHRQ Effectiveness Health Care programs, and others. The CER in the United States may be described at cross-roads or at the beginnings of a scientific era of CER and evidence-based medicine (EBM). However the United States suffers as other countries, including the United Kingdom with its National Health Services (NHS) and National Institute for Health and Clinical Excellence (NICE), with major misunderstandings of methodology, an inordinate focus on methodological assessment, lack of understanding of the study design (placebo versus active control), lack of involvement of clinicians, and misinterpretation of the evidence which continues to be disseminated. Consequently, PCORI and CER have been described as government-driven solutions without following the principles of EBM with an extensive focus on costs rather than quality. It also has been stated that the central planning which has been described for PCORI and CER, a term devised to be acceptable, will be used by third party payors to override the physician’s best medical judgement and patient’s best interest. Further, stakeholders in PCORI are not scientists, are not balanced, and will set an agenda with an ultimate problem of comparative effectiveness and PCORI that it is not based on medical science, but rather on political science and not even under congressional authority, leading to unprecedented negative changes to health care. Thus, PCORI is operating in an ad hoc manner that is incompatible with the principles of evidence-based practice. This manuscript describes the framework of PCORI, and the role of CER and its impact on interventional pain management. Key words: Patient-Centered Outcomes Research Institute (PCORI), comparative effectiveness research (CER), National Institute for Health and Clinical Excellence (NICE), Patient Protection and Affordable Care Act (ACA), Medicare Modernization Act (MMA), American Recovery and Reinvestment Act (ARRA), interventional pain management, interventional techniques, evidencebased medicine, systematic reviews.

Sign in / Sign up

Export Citation Format

Share Document