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The makers of two coronavirus vaccines, Pfizer and its German partner BioNTech, and Massachusetts-based Moderna, have applied to the FDA for emergency use authorization to begin vaccinating Americans. If both companies get authorization, the US government could begin distributing the vaccines before the end of December. The speed of the development and approval of these vaccines is like nothing we’ve ever seen before. It is inspiring to see science move so quickly, but it is instilling some worry in people. How can something developed so quickly be safe? Moreover, will the vaccines be safe for people with bleeding disorders?
 
These aren’t unreasonable questions, especially in a year that has gotten more than the usual mileage out of the term “unprecedented.” However, these questions do have answers.
 
It is true that the Pfizer/BioNTech and Moderna vaccines have been developed very quickly. But the technology behind the vaccines is not new. They both are what are called mRNA vaccines, which function very differently than the types of vaccines used in the past. mRNA vaccines, rather than using a weak or ineffective sample of the virus to trigger antibodies – like in a flu shot – mRNA vaccines instruct your cells to create a harmless protein (called “spike” protein) that your body recognizes as foreign, creating an immune system response. Papers describing this mRNA technology, discovered by Hungarian scientist Katalin Karikó (who oversees mRNA work for BioNTech) were first published in 2005.

Vaccines, as with all medicines and devices regulated by FDA, undergo a rigorous review of the laboratory and clinical data to ensure the safety, efficacy, purity and potency of these products. Vaccines approved for marketing may also be required to undergo additional studies to further evaluate the vaccine and often to address specific questions about the vaccine's safety, effectiveness or possible side effects.

The FDA recognizes the seriousness of the current public health emergency and the importance of facilitating availability, as soon as possible, of vaccines to prevent COVID-19—vaccines the public will trust and have confidence in receiving.

An Emergency Use Authorization (EUA) is a mechanism to facilitate the availability and use of medical countermeasures, including vaccines, during public health emergencies, such as the current COVID-19 pandemic. Under an EUA, FDA may allow the use of unapproved medical products in an emergency to diagnose, treat, or prevent serious or life-threatening diseases or conditions such as infection with the virus that causes COVID-19 disease when certain statutory criteria have been met, including that there are no adequate, approved, and available alternatives.

With this understanding, the vaccines that will likely be available before the end of 2020 will have EUA status and not full FDA approval. This does not mean they are not safe. Even people with bleeding disorders can and should receive one of the vaccines when it is available. Of course, as with all medications, vaccines are no different, people must be monitored for side effects. This will be the shared responsibility of the people who receive the vaccine, their healthcare providers along with the FDA and the U.S. Centers for Disease Control and Prevention (CDC). We should all be in line (with a mask on and six feet between us) to receive the vaccine. I know I will when it's available to me!

There is one possible concern for people with a bleeding disorder who may be considering gene therapy in the future. One drug that is in development by Regeneron will be using an adeno-associated virus to deliver a gene into the body using a nasal spray to create the COVID-19 antibody cocktail, similar to what was used intravenously by the President a few weeks ago. However, this drug is a ways away from clinical testing.

The distribution of the vaccine will be an enormous undertaking, involving both the federal and local governments. It is likely that for many of us, a vaccine will not be available until the spring or even summer of 2021. In the meantime, everyone should get the vaccines that are available that will help protect you against seasonal illness that can cause worse outcomes in patients with COVID-19. If you have not yet done so, get your annual flu shot. People over the age of 19 who haven’t gotten a Tdap (tetanus, diphtheria and pertussis) vaccine should ask their doctor for that vaccine as well.
The CDC is recommending that healthcare workers and residents of nursing homes be the first to receive the new coronavirus vaccines. The recommendation was proposed by an independent panel, the Advisory Committee on Immunization Practices, and approved and adopted by the CDC on December 2.
Physician Commentary
A recent article published in the journal Food Science & Nutrition, “Effects of the dietary approach to stop hypertension (DASH) diet on blood pressure, blood glucose, and lipid profile in adolescents with hemophilia: A randomized clinical trial,” sought to investigate the effect of the Dietary Approaches to Stop Hypertension (DASH) diet on cardiovascular and metabolic risk factors among children with hemophilia. We asked two physicians, Roshni Kulkarni, MD, Michigan State University Center for Bleeding and Clotting Disorders; and Courtney Thornburg, MD, Hemophilia and Thrombosis Treatment Center at Rady Children's Hospital-San Diego, for their comments.
Dr. Roshni Kulkarni:

  1. The current article was a 10-week study of the impact of DASH or dietary intervention on various lab parameters of cardiovascular risk factors including blood pressure. The ages chosen were 10 years to 18 years. The author mentions hemophilia – but looks like on table 2, most were FVII deficiency and FIX deficiency– don’t know how many were hemophilia A subjects.
  2. 10 weeks is insufficient time to see any long-term impact. Were there any physical activity parameters collected? Impact on weight, BMI physical activity, treatment?
  3. What was the severity and baseline characteristics of these patients? Joint disease, ABRs, kidney, heart disease (although doubt if they had it).
  4. Was the blood pressure measured on more than one occasion using a right cuff size – especially in the youngest subjects?
  5. What role does pain play in hypertension and do the new therapies and prophylaxis allow for more activity and thereby reduce obesity, hypertension and improve activity? 

“I think that there should be awareness that there are many “H’s” to hemophilia: hemophilia, HIV, hepatitis, heart disease, hypertension, hematuria, and hemarthroses. We have overcome some such as HIV and hepatitis. But others such as heart disease and hypertension are silent killers and are still prevalent and can be prevented. Early onset of hypertension can lead to heart and kidney diseases and therefore routine monitoring of blood pressure, raising awareness and prevention through recognition, diet and exercise is important and should start at a young age. The role of dietitian as a part of the hemophilia comprehensive teams is crucial and should be emphasized.
Dr. Courtney Thornburg comments:

I agree with Roshni. I am bit surprised that this article was published; perhaps this is why a RCT is published in this journal for Food Science and Nutrition. My guess is that table 2 has a typo and FVII should be FVIII. Also, I agree with Roshni’s statement regarding the many H’s of hemophilia and perhaps we can highlight the role of the dietician as part of the care team.

Prevention and management hypertension is particularly important for individuals with hemophilia, because it is associated with increased risk of intracranial hemorrhage in adults with hemophilia. Hypertension may be related to obesity, hemophilia-related pathophysiology, as well as other causes. Given the risks of hypertension, it is important for Hemophilia Treatment Centers to use appropriate blood pressure screening protocols and age-based norms to evaluate for hypertension. Recommendations for detection and diagnosis are provided by the “NHLBI 4th Report on the Diagnosis, Evaluation, and Treatment of High Blood Pressure in Children and Adolescents.” The NHLBI Report also provides management recommendations including “therapeutic lifestyle changes” with a focus on weight-reduction for associated obesity, limiting sodium intake, and getting regular physical activity.

Dieticians can provide important nutritional recommendations for adolescents and adults with hemophilia. At Rady Children’s Hospital San Diego, a dietician is a key member of our comprehensive care team and meets with patients and family annually. Dietary intake, including supplements, are evaluated. The growth chart with height, weight and BMI percentile is reviewed, including trends over time. Recommendations are provided to limit fats, salt and sugar and to increase intake of water, fruits and vegetables, and calcium/vitamin D. Age-based institutional nutrition and the USDA ChooseMyPlate (ChooseMyPlate.gov) handouts are provided. The dietician works closely with the physician and dietician regarding a treatment plan for healthy living. When indicated, referrals are made for nutrition classes and the Weight and Wellness Center. If hypertension or pre-hypertension is identified then recommendations are made for follow-up with the primary care physician and, in some cases, a pediatric nephrologist. 

The article also highlights the importance of adherence. Strategies to improve adherence with treatment recommendation are true for all aspects of care from clotting factor to physical activity to diet. Close collaboration and shared decision making between the patient and treatment team can guide an individualized plan and promote adherence.
President Trump has issued a number of executive orders on drug pricing. We show which ones could affect people with bleeding disorders.
Sign up for NHF’s Friday webinar series! Each month, we’re bringing you experts discussing vital information on bleeding disorders, the COVID-19 pandemic, research, gene therapy and more. Our next Friday webinar is this Friday, December 11 beginning at 4:00 PM EDT – Be sure to join us!
 
If there is a particular topic important to people with bleeding disorders that you would like us to cover in the next newsletter, please send your suggestions to communications@hemophilia.org.
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