Excellence in Care of Sickle Cell Disease Leads to National Recognition

The National Alliance for Sickle Cell Centers (NASCC) has recognized the University of Miami Miller School of Medicine/Jackson Memorial Hospital sickle cell disease program as one of the nation’s top comprehensive sickle cell centers. The recognition reflects the multidisciplinary approach to care that patients receive, beginning from the time babies with sickle cell disease are identified through newborn screening. This comprehensive care continues throughout the transition years from pediatrics to adult care and into adulthood.

The mission of NASCC is to improve the care and outcomes of people with sickle cell disease by creating a unified national network of high-quality, comprehensive sickle cell centers.

The Miller School sickle cell program is one of 11 adult sickle cell treatment centers and one of eight pediatric sickle cell treatment programs recognized by NASCC. It is the only sickle cell disease adult and pediatric program recognized by the NASCC in Florida. The adult program is led by Thomas Harrington, M.D., assistant professor of medicine, and the pediatric program is led by Ofelia Alvarez, M.D., professor of clinical pediatrics.

The Miller School and Jackson Memorial Hospital have collaborated in the care and study of sickle cell disease since the 1980s, according to Dr. Harrington.

The institutions’ shared focus on sickle cell disease began with pediatrician Charles Pegelow, Jr., M.D.; Don Temple, M.D., Dr. Harrington’s predecessor at Sylvester in hematology; Astrid Mack, M.D., a geneticist and local leader in the sickle cell community; and Daniel Armstrong, Ph.D., director of the Mailman Center for Child Development of the Miller School.

Over the past 20 years, Dr. Harrington and Dr. Alvarez have built the programs for adult and pediatric patients. Today the Miller School sickle cell disease team of specialists treats 300 adults and 450 children with the disease.

“Besides providing excellent care, we have participated in almost all major clinical trials in sickle cell disease, providing cutting-edge clinical research for both children and adults,” said Dr. Alvarez. “We are also involved in networking with other institutions throughout Florida and in the Caribbean.”

Complex disease requires prompt diagnosis, comprehensive care

A group of inherited red blood cell disorders stemming from a hemoglobin mutation, sickle cell disease affects an estimated 100,000 Americans. According to the Centers for Disease Control, it occurs in about one out of every 365 Black or African-American births and about one in every 16,300 Hispanic-American births. In the United States, sickle cell disease is diagnosed at birth through newborn screening, which allows for early identification and treatment, decreasing complications and preventing early mortality.

Alex’s Place, the pediatric hematology-oncology clinic within Sylvester Comprehensive Cancer Center, is the medical home for children with cancer and hematological disorders such as sickle cell disease and hemophilia.

A referral center for newborns diagnosed with sickle cell disease, Alex’s Place also works closely with the Miller School’s Stroke Division, as stroke is one of the worse consequences of the disease in children.

Sickle cell treatments range from infusions and blood transfusions to bone marrow transplantation, a potential cure for the most severe cases, according to Dr. Alvarez. New therapies are being developed to improve the health and quality of life of patients with the disease.

According to Dr. Harrington, sickle cell disease is largely misunderstood even by members of the medical community, many of whom see sickle cell care primarily in terms of management of pain and anemia.

“Because sickle cell disease causes severe pain, many patients need strong narcotics for pain,” said Dr. Harrington. “As a result, they are often mistreated and mislabeled as ‘drug-seekers.’ This is demeaning and dangerous, because poor experiences can make patients avoid getting the urgent care they need. Depression is extremely common.”

In reality, sickle cell disease is a chronic vascular condition that damages virtually every organ.

“Chronic kidney, lung, heart, and brain injury, as well as peripheral vascular ulcers, lead to very poor quality of life and a much shorter lifespan for sickle cell patients,” Dr. Harrington said. “We have specialists in all heart, lung, and kidney diseases, as well as in orthopedics, neurosurgery, dermatology, and psychology, who have worked with us for years to manage these issues as a team.”

This focus on high-quality, comprehensive care for sickle cell disease is much needed. Patients with sickle cell disease have less access to comprehensive care than people with genetic disorders such as hemophilia and cystic fibrosis, according to the CDC.

“Unfortunately, patients with sickle cell disease and their families have historically had a degree of mistrust of the health care system,” noted Gerald A. Soff, M.D., director, General Hematology Service, University of Miami Health System and Sylvester Comprehensive Cancer Center. “This contributes to a less-than-ideal quality of care.”

Getting connected with a comprehensive center is known to improve sickle cell disease patient outcomes, said Maya Bloomberg, M.S.N., A.P.R.N., who works with Dr. Harrington and is a family nurse practitioner in Sylvester’s Division of Hematology/Oncology.

“The racial and health disparities seen in sickle cell make it a highly stigmatized disorder that receives very little attention and funding compared to other rare conditions with lower prevalence,” Bloomberg said. “Our recognition by NASCC as a comprehensive center validates to patients that their condition matters.”

The sickle cell disease program at the Miller School provides a vital focal point for patients and their families, minimizing their need to go to emergency departments. This, noted Dr. Soff, makes the patient experience more comfortable while reducing complication rates and costs of care.

“We have built a long-term warm and trusting relationship with 800 sickle cell patients and their families, to whom we provide access to the very latest therapies – some recently FDA-approved, others available in clinical research programs,” Dr. Soff said. “This trust is essential to provide the best care and reduce the hesitation many patients and families have about participating in clinical research studies that could help individual patients and improve sickle cell disease treatment in general.”

Dr. Alvarez noted that the NASCC recognition is an important step in the Miller School’s goal to raise funds to support its mission to improve the lives of individuals with sickle cell disease, build community awareness, expand clinical research, and advocate for policies that will increase patient access to comprehensive care and new therapies.

Tags: Dr. Gerald A. Soff, Dr. Ofelia Alvarez, Dr. Thomas Harrington, Maya Bloomberg, National Alliance for Sickle Cell Centers, sickle cell disease, Sylvester Comprehensive Cancer Center, UM/Jackson