Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity

Abstract BACKGROUND: Sickle cell disease (SCD) and cystic fibrosis (CF) are rare inherited disorders of similar severity. Disparities in funding between these two diseases have been long recognized and likely contribute to limited access to care and treatments for sickle cell disease. Private investment in therapeutics for these and other orphan diseases has greatly increased in the past ten years. We hypothesized that these increased private expenditures may help correct disparities in research publications, clinical trials, and FDA approval of new therapies. METHODS: We compared funding and research output for SCD and CF between 2008-2012 versus 2013-2017. We estimated disease-specific funding using NIH Research Portfolio Online Reporting Tools and the Form 990 financial reports for foundation expenditures of multiple organizations dedicated to each disease from 2008-2016. We developed a comprehensive search strategy to identify relevant publications in PubMed and new postings of US based interventional clinical trials by disease. In addition, we reviewed FDA drug approvals and new indications for each disease from 2008-2017. RESULTS: Average annual NIH funding per affected individual was 3.4-fold greater for CF than SCD from 2008 to 2016. Between 2008-2012, private foundation funding was 161-fold greater for CF than SCD. Between 2013-2016, private funding was 971-fold greater for CF than SCD. There were 1.8 times as many PubMed publications for CF compared to SSD. There was no significant difference in PubMed publications between the two time periods. There was a significant increase in interventional clinical trials for SCD between 2013-2017, with the largest increase coming from university/philanthropic funded trials. However, CF has significantly increased FDA drug approvals of both novel compounds and novel indications. CONCLUSIONS: Although the US prevalence of SCD is three times greater than CF, the amount of federal and private foundation research funding is magnitudes greater for CF. Foundation funding for CF benefited significantly from revenue based on the successful development of targeted therapies. However, despite the significant funding difference, the number of clinical trials for SSD are comparable to CF and have increased over the past few years. Research productivity as measured by articles indexed in PubMed, and new drug approvals remain substantially higher for cystic fibrosis despite greatly increased industry investment in orphan diseases. Disclosures Strouse: Global Blood Therapeutics: Consultancy.

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Factors associated with undernutrition among children with sickle cell disease attending the sickle cell clinic in Mulago National Referral Hospital, Uganda

10.21203/rs.2.12659/v2 ◽

2019 ◽

Author(s):

Rosta Asiimwe ◽

Rornald Muhumuza Kananura ◽

Richard Kajjura ◽

Adoke Yeka

Keyword(s):

Sickle Cell Disease ◽

Sickle Cell ◽

Older Age ◽

Referral Hospital ◽

Childhood Mortality ◽

Mulago Hospital ◽

Cell Disease ◽

Factors Associated ◽

Mulago National Referral Hospital ◽

National Referral Hospital

Abstract Background Sickle cell disease (SCD) is among the neglected non-communicable diseases, which significantly contributes to early childhood mortality. In Uganda, over 20,000 children are estimated to be sicklers. Undernutrition is common among children with SCD and contributes to increased morbidity and mortality. There is paucity of data on prevalence of undernutrition and associated factors in Uganda. Objective To assess the extent of undernutrition and related factors among children aged 5-12 years with SCD attending the sickle cell clinic at Mulago hospital, Uganda. Methods A total of 263 children with SCD attending the sickle cell clinic at Mulago National Referral hospital were recruited consecutively between May and June 2017. The nutritional status of the children was assessed by weight-for-age, BMI-for-age, and height-for-age z-scores calculated using STATA in accordance with WHO 2007 growth standards. Binomial regression was conducted to assess the predictors of undernutrition. Results About 20.2%, 11.4%, and 13.7% of the children were underweight, wasted and stunted respectively. Wasting was significantly associated with older age (10-12 years) (AOR=4.20, CI=2.18-8.10) and living in a female headed household (AOR=0.43, CI=0.19-0.99). Stunting was significantly associated with older age (10-12 years) (AOR=2.90, CI=1.39-6.06). Underweight was significantly associated with older age (10-12 years) (AOR=2.23, CI=1.05-5.16). Conclusion Underweight, wasting and stunting were prevalent among children with SCD attending Mulago hospital. The factors associated with undernutrition were older age and living in a female headed household.

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Red blood cell alloimmunization in sickle cell disease: pathophysiology, risk factors, and transfusion management

Blood ◽

10.1182/blood-2011-11-327361 ◽

2012 ◽

Vol 120 (3) ◽

pp. 528-537 ◽

Cited By ~ 201

Author(s):

Karina Yazdanbakhsh ◽

Russell E. Ware ◽

France Noizat-Pirenne

Keyword(s):

Risk Factors ◽

Sickle Cell Disease ◽

Blood Cell ◽

Sickle Cell ◽

Red Blood Cell ◽

Cell Disease ◽

Factors Associated ◽

Life Threatening ◽

Red Blood Cell Transfusions ◽

Transfusion Reactions

Abstract Red blood cell transfusions have reduced morbidity and mortality for patients with sickle cell disease. Transfusions can lead to erythrocyte alloimmunization, however, with serious complications for the patient including life-threatening delayed hemolytic transfusion reactions and difficulty in finding compatible units, which can cause transfusion delays. In this review, we discuss the risk factors associated with alloimmunization with emphasis on possible mechanisms that can trigger delayed hemolytic transfusion reactions in sickle cell disease, and we describe the challenges in transfusion management of these patients, including opportunities and emerging approaches for minimizing this life-threatening complication.

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