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Sickle Cell Patients Confront A ‘fight For Everything’ — Even Attention

CHICAGO — The day before he turned 30, Marqus Valentine panicked. “I was so frightened for midnight to come rolling around because I was like, ‘This is it. Tomorrow’s my last day on Earth,’&rdquo.

Valentine has sickle cell disease, an inherited blood disorder, and his doctors had warned him throughout his life that he was not likely to make it into 30.

As have four , that birthday passed without occasion. Still, rsquo, Valentine &disease has left him severely disabled. “Instead of 34, I feel as I’m rdquo, & 68; he explained.

He sleeps in a hospital bed on the floor of his parents & rsquo; house wants a hip replacement and utilizes a portable oxygen tank — he is in too much pain to make it up the stairs. And & ldquo still participates:;I could awake in the morning, brush my teeth, eat breakfast and by the time afternoon comes rolling around, I could be dead. ”

Valentine has watched as one powerful advocacy effort after another brought attention and resources to other disorderss disease. But little is learned about cell. While life expectancy for sickle cell patients has diminished, with other treatments and drug development, the past couple of decades have improved over.

To sickle cell patients and their families — efforts to fight the disease appear underfunded slow or ineffective, perpetuating century-old disparities.

“You feel as the baldheaded stepchild nobody cares about,” Valentine said from his bed at Edward Hospital in Naperville, Ill., while recovering from one of the bimonthly blood transfusions. “What about us? We’re here also. ”

Sickle cell disease affects an estimated 100,000 people in the U.S., resulting in chronic pain, multi-organ failure and stroke. With costs to treat the disease soaring beyond $1 billion efforts are afoot to improve many patients. But obstacles are faced by Every development:

►In July, the FDA approved the first new drug to treat sickle cell in just two years. Yet specialists say this month Endari, expected to hit the market, will have limited advantage. And insurance companies are balking at covering it. Several, such as the Valentines’ Blue Cross Blue Shield’s Federal Employee Program, will cover Endari just if patients have “neglected” at other treatments, such as blood transfusions and hydroxyurea (the only other medication available to treat sickle cell).

►Physicians institutions are working to disseminate guidelines to improve care and reduce discrimination against sickle cell patients, frequently assumed to be drug addicts when they visit emergency rooms in acute pain, due to their misshapen red blood cells flushing the vessels and cutting off oxygen to joints and organs. Still, a dearth of research on cell makes it tricky to compose evidence-based protocols.

►Legislation in Congress to finance research and therapy, postponed since 2009, is finally moving from committee. However, the bill would provide just $4 million — less than half its initial funding level.

Most sickle cell patients fight to access even the most basic care. Patients receive misdiagnosed, and ER physicians understand how to cure them and sent home, said Patricia Kavanagh, a physician and ER physician at Boston Medical Center. “What we know about sickle cell disease in med school is covered in 20 minutes. ”

Almost 40%  of sickle cell patients need to be readmitted to the hospital within 30 days — more than for another identification, according to the government’s Agency for Healthcare Research and Quality.

“It is appalling. This country should be ashamed of itself,” stated Valentine’s mother, Francesca Valentine, a registered nurse for 35 years who has been an activist for sickle cell patients. “I’m baffled that in 2017, we’re still not treating the disease based on science, and we still manage racism and stigma and inaccurate details. ”

In 2014, part of the National Institutes of Health, the National Heart, Lung, and Blood Institute, put out guidelines for doctors, and the protocols were endorsed by 11 organizations. But protocols are only as good as the science supporting them, and “the signs for most of the guidelines isn’t strong,” because there haven’t been enough research on the disease, said Rosalyn Stewart, a Johns Hopkins physician and an investigator on a national grant to improve care for sickle cell patients.

Sickle cell funding pales in comparison to other ailments. Cystic fibrosis, which affects 30,000 people in the U.S., by way of instance, gets seven to 11 times more funding per patient than sickle cell disease, according to a 2013 study from the journal Blood. The ALS barrier in 2014 raised $115 million for roughly 20,000 patients at the U.S.

Some new sickle cell studies have been including eight financed from the Patient-Centered Outcomes Research Institute. However one major issue is that no data registry is present for sickle cell disorder, stated director of the sickle cell program at the Centers for Disease Control and Prevention, Mary Hulihan.

“We don’t have basic information such as how a lot of people in the U.S. have sickle cell disease or where they are getting their healthcare,” Hulihan explained.

Regardless of the setbacks, patients and experts remain optimistic that the treatment of sickle cell disease may gain attention and momentum.

But if rsquo; t pay Endari for her son & insurance doesn, Francesca Valentine stated, her and her husband could want jobs to afford the medication. If needed, by writing letters explaining the science, she plans to challenge her insurer.

Like this disorder must struggle for everything, & ldquo; It & rsquo; s. We should not have to fight for all,&rdquo.