NOSE Study Results Are In!

 

PUBLICATION: Journal of American Medical Association; JAMA. 2016;316(9):943-951. doi:10.1001/jama.2016.11724

 

We are very pleased to report the first multi-center clinical study on nosebleed treatment has been published and released in the Journal of American Medical Association: “Effect of Topical Intranasal Therapy on Epistaxis Frequency in Patients with Hereditary Hemorrhagic Telangiectasia a Randomized Clinical Trial” on September 6, 2016.

This is the first randomized double blind placebo controlled Phase II Clinical trial ever documented! This study has propelled other studies forward, such as the Avastin Epistaxis study at Stanford University and the Paznopanib trial with Glaxo Smith Kline Pharmaceutical Company.

Results

120 patients received topical therapy of either Avastin 1% (4mg/d), Estriol 0.1% (0.4mg/d), Tranexamic acid 10% (40mg/d) or placebo (0.9% saline) for 12 weeks to assess the risks and benefits for treatment of moderate to severe nose bleeding. As you know all too well, interventional techniques such as laser and cautery have provided temporary help, but the need for better therapeutic approaches in managing nosebleeds is desperately needed.

The study showed treatment with topical therapy is safe, well tolerated and improves nosebleed severity in most patients. Though the study didn’t show any impact on nosebleed frequency, patients on Avastin showed a borderline reduction in nosebleed duration in comparison with the tranexamic acid and placebo over the 24 weeks.

Variables

As HHT patients do not routinely irrigate the nose prior to the administration of drug therapies (which might improve absorption of medication), we do not know if this procedure impacted the results. Additionally, it may be that a higher concentration of the Avastin (bevacizamab) could have produced greater improvement.

Conclusion

This study concluded simple treatment with nasal saline spray twice daily is beneficial to many patients. The addition of tranexamic acid, estriol or bevacizumab does not seem to offer significant advantage over plain saline. However, if you are currently using one of these medicated sprays, do not discontinue before speaking with your doctor.

GOSSAGE SIGNATURE FOR LETTER

 

 

James Gossage

Medical Director

Marianne Clancy signature

 

 

Marianne Clancy

Executive Director

 

Cure HHT Launches New York Center

New Center Hospital Photo

24th HHT Center of Excellence in North America

NewYork Presbyterian/Columbia University Medical Center

New York, NY

“Two years ago, we made a commitment to New York and New Jersey families that we would work very hard to encourage a team of experienced physicians, with our guidance and a HHT mentor center, to come together as an HHT Center of Excellence,” said Executive Director Marianne Clancy. “I am so happy to see this work become a reality. I am confident this superb team, which I met during a site visit, will provide outstanding care. We look forward to working closely together in the years to come."

nyc

NewYork Presbyterian/Columbia University Medical Center is the only facility in New York that specializes in the diagnosis, evaluation and treatment of HHT. The newest HHT Center offers compassionate, family-centered multidisciplinary care, led by Dr. Sophie Chheang, a dynamic and action-oriented physician. At the NewYork Presbyterian/Columbia University HHT Center, each patient is treated by HHT-knowledgeable physicians who are compassionate and devoted to maintaining the highest standards of care. Dr. Chheang’s team includes a full team of pediatric specialists lead by Dr. Emilio Arteaga-Solis.

Chheang, Sophie MDDr.Chheang is a board certified radiologist at NewYork Presbyterian and an assistant professor of radiology at Columbia University Medical Center. After graduating from the New York University School of Medicine, she completed her Diagnostic Radiology residency at the University of California, Los Angeles. Dr. Chheang then finished her Interventional Radiology fellowship at NewYork-Presbyterian/Weill Cornell Medical Center,which included additional training at the Memorial Sloan Kettering Cancer Center.In addition to English, Dr.Chheang speaks Khmer and Spanish. She is Board certified with the American Board of Radiology.

 

Specialties at NewYork Presbyterian/Columbia University HHT Center

Cardiology

Dermatology

Genetics/Genetic Counseling

Gastroenterology

Hematology

Interventional Radiology

Neuro-interventional Surgery

Neurosurgery

Otolaryngology (ENT)

Pediatrics

Pulmonology

 

To Make an Appointment

Adult Appointment: Contact Helen Pena-Chacon at 212-305-7094

Pediatric Appointment: Contact Emilio Arteaga-Solis, MD, PhD at 212-305-5122

HHT and Pregnancy

Most women with HHT can experience normal pregnancy and delivery with the same amount of risk as women in the general population. Though HHT pregnancies are sometimes considered “high-risk," this is not usually the case. While women with more concerning HHT symptoms can benefit from a “high-risk” clinic approach, women who experience minimal symptoms may not need it. The key is to consult with an HHT expert to help make the best decisions for you and your baby.

Remember that HHT affects each person differently, and symptoms are known to change over a lifetime. How HHT affects your body should guide your pregnancy planning and care. For example, if you only experience telangiectasia in the nose and have no abnormal blood vessels in the organs, there's no reason to think your pregnancy or delivery is high-risk. On the other hand, if you have lung AVMs, then several steps and precautions need to be taken to minimize risk, and in some cases this may be considered a high-risk pregnancy.

Here's What You Need to Know

Myth #1:  Women with HHT are at risk of excessive bleeding from the uterus during or after delivery.

There is no evidence of this. In fact, women with HHT are no more likely to bleed excessively at delivery or after delivery than women in the general population.

Here’s why: People with HHT don’t have a clotting disorder. People with HHT bleed from parts of the body where they have abnormal blood vessels, not because of a regular injury, trauma or cut. The uterus is not a typical place for AVMs to occur in HHT, so there is no reason to expect women with HHT to bleed with delivery.

Myth #2: Women with HHT can’t have an epidural.

Rest easy  - this is not the case. Most women with HHT decide to have an epidural, or give themselves the option to have one available in the moment. There is no evidence of increased risk of complications. We'll break it down for you.

Why is there concern? Many anesthetists and other doctors are worried about the risks of epidural anesthesia in women with HHT, specifically about puncturing a spinal AVM when they insert the epidural needle into the women’s lower spine. Approximately 1 percent of HHT patients have AVMs in the spinal canal, but the majority of HHT spinal AVMs are higher on the spinal canal than where the epidural is inserted.

No case has ever been reported in medical literature of an epidural causing spinal bleeding in HHT. Complications of spinal AVMs are exceedingly rare in adults (most are discovered during childhood). In fact screening for spinal AVMs isn't routinely recommended in adults with HHT (unlike screening for lung and brain AVMs) given how rare they are in adults. In other words, if there is a risk, it's so low no one has been able to document a case or quantify the risk. However, there is no study “ruling out” risk of epidural in HHT - so we call it a “theoretical” risk.

If a pregnant woman with HHT wants the option of an epidural, she should get the option. Meet with the anesthetist prior to delivery and have a discussion about all the risks,“theoretical” or otherwise. Many anesthetists will agree to proceed if the woman understands the risks.

Myth #3: Women with HHT should have a cesarean section to avoid bleeding and other HHT complications with labor and delivery.

There is no evidence to support the C-Section as a routine approach for HHT patients. We've already discussed the myth of excessive bleeding in #1 so let us continue to other assumed complications.

The second concern is a brain AVM rupturing when pushing at delivery. There is no evidence to support this worry, or that cesarean section is a safer alternative to vaginal delivery in people with brain AVMs. However, this situation needs to be handled on an individual basis.  Screening for brain AVMs can be performed in early third trimester with an unenhanced MRI of the brain. If the MRI is negative, as it is for 90 percent of women with HHT, then there's no reason to be concerned.

If you do have a brain AVM, your case is considered high risk and needs to be discussed with a brain AVM expert and obstetrician. If a brain AVM is diagnosed, the usual recommendation is to treat within two or three months after delivery. As such, since treatment will be delayed anyway, some women opt to postpone the brain MRI until after delivery. This is not unreasonable, but may leave you and your physicians unnecessarily worrying about the possibility of brain AVMs and a false “high-risk” pregnancy.

There is also no evidence that a treated lung AVM is more likely to rupture and bleed during vaginal delivery. However, if you have an untreated lung AVM your pregnancy should be considered high risk. Screening and preventative treatment for lung AVMs should be done prior to pregnancy, when possible. If you are already pregnant, lung AVM treatment during Trimester 2 or 3 with embolization should be considered, but only at an expert HHT center. If you have lung AVMs that have been successfully treated (and recently reassessed), then lung AVMs are not a concern for pregnancy and delivery.

Summary

For most women with HHT, the main problem is nosebleeds, and though bothersome, this is not often a major concern during pregnancy. Some women with HHT have a more extensive issues, with AVMs in the brain or lungs, which may be more concerning. However, with the right screening, treatment and surveillance, most women with HHT can have normal pregnancy and delivery, with no more risk than women without HHT. Any patient with lung AVMs (treated or untreated) should follow pulmonary AVM precautions, including antibiotics before any procedures that can cause bacteria in the blood as well as using an air filter (bubble trap) any time there is an intravenous access.

References

  1. Faughnan, M.E., et al., International guidelines for the diagnosis and management of hereditary haem- orrhagic telangiectasia. J Med Genet. 48(2): p. 73-87.
  2. De Gussem, E.M., et al., Outcomes of Pregnancy in HHT(Abstract). Hematology Meeting Reports, 2009. 3(4): p. 10.
  3. Gershon, A.S., et al., Transcatheter embolotherapy of maternal pulmonary arteriovenous malformations during pregnancy. Chest, 2001. 119(2): p. 470-7.
  4. Shovlin, C.L., et al., Medical complications of pregnancy in hereditary haemorrhagic telangiectasia. QJM, 1995. 88(12): p. 879-87.
  5. Shovlin, C.L., et al., Estimates of maternal risks of pregnancy for women with hereditary haemorrhagic telangiectasia (Osler-Weber-Rendu syndrome): suggested approach for obstetric services. BJOG, 2008. 115(9): p. 1108-15.
  6. Wain, K., et al., Hereditary hemorrhagic telangiecta- sia and risks for adverse pregnancy outcomes. Am J Med Genet A, 2012.
  7. http://www.nichd.nih.gov/health/topics/high_risk_ pregnancy.cfm