New Research Led by Johns Hopkins All Children’s Hospital Finds Shorter Treatment Effective for Young Patients with Blood Clots

01/11/2022

Neil A. Goldenberg, M.D. PhD.
Neil A. Goldenberg, M.D. PhD.

St. Petersburg, Fla. – January 11, 2022 – New research led by Johns Hopkins All Children’s Hospital, now published in the Journal of the American Medical Association (JAMA), has found a practice-changing advancement in children and young adults with blood clots. After collecting 13 years of data from patients under 21 years old from around the world, findings revealed that a six-week course of treatment with anti-clotting medications (called “anticoagulants”) for acute, provoked blood clots in patients under 21 years old was just as effective, and at least as safe, as the standard anticoagulant treatment of three months.

Nearly two decades ago, during his residency training, Neil Goldenberg M.D., Ph.D., professor of pediatrics and medicine at Johns Hopkins University and director of the Thrombosis Program in the Johns Hopkins All Children’s Cancer and Blood Disorders Institute, came up with the idea for an innovative clinical trial to determine the optimal duration of treatment with anticoagulant medications in young patients with blood clots – also known as deep vein thrombosis and pulmonary embolism, or venous thromboembolism (VTE). It then became part of his life’s work to improve the treatment and prevention of VTE in children and young adults, through research that has spanned from the laboratory to the bedside and community.  

“Previous treatments relied on trials from the 1990s in older adult VTE patients, so it was important for us to find a better solution for the growing number of infants, children teens and young adults affected by VTE,” said Dr. Goldenberg, who also serves as associate dean for research and director of the Johns Hopkins All Children’s Institute for Clinical and Translational Research. “Shorter treatment should not only result in significant savings in health care costs, but more importantly, is expected to improve quality of life for patients and families. Being on anticoagulant medications, what we used to call ‘blood thinners’, means having to worry about and take significant precautions against bleeding complications. Coming off of anticoagulant medications in half the amount of time as in the past means being able to return to normal routines, activities and sports much sooner.”

VTE affects approximately 1 in 200 hospitalized children, and 1 in 50 children hospitalized with critical illnesses, making it one of the most common hospital-acquired pediatric health conditions. In addition, for more than the past year, children’s hospitals have been seeing a spike in VTE attributable to acute COVID-19 infection, especially among those who develop the multisystem inflammatory syndrome in children (known as MIS-C).

The landmark randomized clinical trial — called Kids-DOTT (Duration of Therapy for Thrombosis) — included 50 collaborating institutions from throughout the United States, Canada, Europe and Australia, and involved more than 400 patients under 21 years old. For the past five years, the study has been funded by two U01 research grants (each providing approximately $3 million in support) from the National Institutes of Health (NIH) and National Heart, Lung, and Blood Institute (1U01HL130048). Under the current grants, further analyses and reports will address additional key questions regarding treatment and outcomes in young patients with VTE. Dr. Goldenberg and his team are also applying for new grants to conduct follow-up studies on how the practice-changing findings of the Kids-DOTT trial are adopted into clinical care globally, and to monitor the degree to which the favorable outcomes measured in Kids-DOTT continue to be observed in subsequent real-world experience.



About Johns Hopkins All Children’s Hospital
Johns Hopkins All Children’s Hospital in St. Petersburg is a leader in children’s health care, combining a legacy of compassionate care focused solely on children since 1926 with the innovation and experience of one of the world’s leading health care systems. The 259-bed teaching hospital stands at the forefront of discovery, leading innovative research to cure and prevent childhood diseases while training the next generation of pediatric experts. With a network of Johns Hopkins All Children’s Outpatient Care centers and collaborative care provided by All Children’s Specialty Physicians at regional hospitals, Johns Hopkins All Children’s brings care closer to home. Johns Hopkins All Children’s Hospital consistently keeps the patient and family at the center of care while continuing to expand its mission in treatment, research, education and advocacy. For more information, visit HopkinsAllChildrens.org.