Marisol Betensky, M.D., M.P.H., likes the challenge. She likes analyzing the options and possibilities. Hematology-oncology causes her mind to click into motion.

“The first thing that attracted me was the science, particularly hematology,” says Betensky, who joined Johns Hopkins All Children’s Hospital in St. Petersburg, Florida, as a hematology-oncology specialist in 2016. “I find that it takes a lot of thinking about the patient, and it's like putting the pieces of a puzzle together in order to get a diagnosis. It’s intellectually very challenging.”

Now, Betensky hopes to find some missing pieces of the hematology puzzle, particularly those related to thrombosis — blood clots in blood vessels — in patients with sickle cell disease (SCD).

Betensky, also an assistant professor of Pediatrics in the Division of Hematology at Johns Hopkins University School of Medicine, recently received a five-year, $959,949 Mentored Patient-Oriented Research Career Development Award from the National Institutes of Health (NIH). Known as a K23 award, the grant will support a portion of Betensky’s salary and research, including special blood sample analysis, in an NIH pathway designed to support individuals with a clinical doctoral degree who have made a commitment to patient-oriented research.

Driving Her Interest

Betensky says she developed an interest in thrombosis by being in the right places at the right times. She clicked with mentors who had special interest in thrombosis, especially Neil Goldenberg, M.D., Ph.D., a professor of Pediatrics and Medicine at Johns Hopkins University (JHU) School of Medicine, associate dean for research at Johns Hopkins All Children’s and director of the Johns Hopkins All Children’s Institute for Clinical and Translational Research, who leads the hospital’s pediatric thrombosis program.

“As I started getting involved in thrombosis under his mentorship, I realized that this is one of the conditions that is becoming so relevant in kids, particularly hospitalized kids,” she says.

As medical care has improved to save more children’s lives, an unintended consequence is they may experience longer hospital stays, which can lead to thrombosis, which is the second-leading complication among hospitalized children.

Sickle cell disease is among those conditions where medical care has made great strides. It is an inherited blood disorder that interferes with red blood cells’ ability to deliver oxygen to the tissues of the body. Ninety percent of the about 100,000 people in the United States who have sickle cell disease are of African descent. Individuals with sickle cell often have excessive blood clotting (hypercoagulability). Venous thromboembolism (VTE) is a condition that occurs when a blood clot forms in a vein. 

Despite this known association, VTE has remained an understudied complication of pediatric sickle cell disease, leading to a scarcity of data on VTE outcomes and hindering the optimal management of VTE in children with sickle cell disease.

Through a multicenter prospective-retrospective cohort study with parallel biospecimen banking, Betensky hopes to develop critical knowledge to better predict long-term outcomes of VTE in this population.

“Before medicine got so much better at treating very sick kids, we didn’t see many blood clots in children,” says Betensky, also medical director of International Patient Services at Johns Hopkins All Children’s. “But now we’re helping them to live longer lives, and we’re seeing some complications. There's a lot that we still need to do in order to understand how to prevent kids from clotting, and what the best treatments are.”

A Guiding Hand

K23 awards are intended to encourage mentorship and career development of junior faculty. Goldenberg has served as a primary research and career development mentor for Betensky since she joined the Johns Hopkins All Children’s medical staff, and now serves in this role under the K23 award.

“He’s very generous with opportunities,” Betensky says. “He constantly gets offered things to do and always is thinking about passing them along to his mentees. He has opened door after door introducing me to people. He always has his mentees in mind wanting them to get the credit or the spotlight, which is not common in all mentors.”

Goldenberg, also the Perry Family Endowed Professor in Clinical and Translational Research, credits Betensky’s determination and skill for her achievements.

“Dr. Betensky’s research and clinical expertise in pediatric thrombotic disorders have each garnered increasing international recognition over the past several years since her recruitment to the Johns Hopkins All Children’s campus as a JHU faculty member,” Goldenberg says. “She is now the second clinician-researcher at Johns Hopkins All Children’s to receive a prestigious NIH Career Development Award in this past year.

“The ecosystem for early-career investigators at Johns Hopkins All Children’s is really exciting and continues to grow, also benefiting from and enhancing our collaborations with colleagues on the Johns Hopkins Medicine campuses in Baltimore.

“Dr. Betensky and her fellow early-career clinician-researchers at Johns Hopkins All Children’s are tremendously talented, inspire so many others including myself, and are driven by a passion to make a positive impact on the lives of not only the children they care for, but also the children around the world whose care is advanced through their research.”

Betensky also has mentoring relationships with Cassandra Josephson, M.D., director of the Johns Hopkins All Children’s Cancer & Blood Disorders Institute and professor (PAR) in Oncology and Pediatrics in the Johns Hopkins University School of Medicine, and Vera Ignjatovic, Ph.D., assistant director for translational research in the Johns Hopkins All Children’s Institute for Clinical and Translational Research and professor of pediatrics (PAR) in the Johns Hopkins University School of Medicine.

“Dr. Betensky is an extremely driven and passionate physician-scientist,” Josephson says. “I am thrilled for her to achieve this milestone in her path towards becoming an independently funded researcher who will be making a significant contribution in such an understudied and critical area for this patient population. I am proud to a part of her talented and accomplished mentorship team.”

“It’s impressive the type of research organization Dr. Goldenberg and others have built at Johns Hopkins All Children’s,” Betensky says. “You don’t realize until you’re doing multicenter trials that most other institutions don’t have these resources, all these people to help. We have a team whose job is to help me start the study, and then I have another team that helps me conduct the study. It’s so much support.

“This grant is fantastic. I feel it’s going to help us fill an important knowledge gap in the fields of pediatric VTE and sickle cell disease, which at the end of the day will lead to the most important thing — better treatment for our patients.”