Connective Issues Fall 2018

MEDICAL INFORMATION

MEDICATION UPDATE By Josephine Grima, PhD, Chief Science Officer, The Marfan Foundation Fluoroquinolones are a class of antibiotics that are commonly used to treat a variety of illnesses, such as respiratory and urinary tract infections. These medicines include ciprofloxacin (Cipro), gemifloxacin (Factive), levofloxacin (Levaquin), moxifloxacin (Avelox), norfloxacin (Noroxin), and ofloxacin (Floxin). There has been conflicting results as to whether this class of antibiotics can result in retinal detachment or aortic aneurysms and dissections. In May 2017, the FDA updated their information about fluoroquinolones indicating that published studies currently do not support reports that these medicines may result in New research studies recently pub- lished in JAMA Surgery have found new evidence in an aortic aneurysm and dissection mouse model that demonstrates the addition of ciprofloxacin to this vulnerable mouse model is associated with severe aortic destruction, increased incidence of aortic aneurysm, dissection and death. The results of this study are being transferred to the FDA for further consideration. Currently, there is evidence that these medicines are associated with disabling and potentially per- manent side effects of the tendons, muscles, joints, nerves, and central nervous system. At this time, the Foundation’s Professional Advisory Board suggests that the general FDA warnings be adhered to. Therefore, fluoroquinolones should be reserved for use in patients who have no other treatment options unless the benefits of the drug will outweigh the risks for serious bacterial infections. retinal detachment or aortic aneurysms and dissections.

NEONATAL MARFAN SYNDROME New Resource Available

NATHAN AND LYNETTE ELAMWITH ANDY, WHO HAS NEONATAL MARFAN, AND THEIR NEWBABY, LUKE.

condition. A prior definition that required death by 2 years of age caused a dramatic and obligate pessimistic outlook for this disorder. It is now clear that some children who would meet anyone’s definition of neonatal or infantile Marfan syndrome in infancy are surviving and thriving in later childhood and young adult life. The danger in neonatal Marfan syn- drome is severe valve dysfunction with consequent heart failure as this can lead to early death. Reports of aortic dissection in infancy or early childhood in Marfan syndrome are exceedingly rare. The outlook for children with infantile onset and rapid progression of Marfan syndrome is likely to continue to improve with further advances in the medical and surgical management of cardiovas- cular manifestations. The new fact sheet further describes neonatal Marfan syndrome. It explains: • Clinical diagnosis • Genetic testing • Treatment • Unique surgical considerations • Causes To download the new fact sheet, please visit the patient and family resource section of our website.

Now available on the Foundation’s website, marfan.org, is a new fact sheet on neonatal Marfan syndrome. This diagnosis is very different than Marfan syndrome in that it has early onset and rapidly progressive features, most seriously related to the heart, lungs, and airways. Significant heart issues are evident in early infancy, and there is rapid progression during childhood. Poor feeding and breathing issues, as well as pronounced outward features of Marfan (e.g., long arms, legs, fingers, joint laxity, and contractures), are also evident early on. “Finding out that our baby was born with neonatal Marfan syndrome was very overwhelming for our family, especially since, like many families with neonatal Marfan kids, our son was a spontaneous mutation and we had previously never heard of Marfan syndrome,” said Lynette Elam of Andover, MA. “It’s been an ongoing journey determining which specialists we need to comprise our son’s care team. The Foundation’s new fact sheet will be so helpful for families, especially those with kids recently diagnosed.” Life expectancy in neonatal Marfan syndrome depends on inconsistent and somewhat arbitrary definitions of the

12 Marfan.org