Hemophilia and Thrombosis Treatment Center - Pediatric

Overview

The Mass General Hemophilia and Thrombosis Center (HTC) offers comprehensive care for children and adults with bleeding disorders including hemophilia A, hemophilia B, rare clotting factor deficiencies, and von Willebrand disease. We also care for patients with platelet dysfunction and thrombocytopenia. The HTC provides comprehensive care to children, adolescents and adults with complex inherited or acquired thrombotic disorders, including deep venous thrombosis, pulmonary embolism and pediatric stroke and TPP (for children without symptoms whose parents or other close relatives have suffered a major thrombotic event, we offer evaluations of and counseling for inherited risk factors.)

Mission

Our pediatric and adult medical teams work cooperatively to treat patients throughout their lifespan, providing exceptional care from birth through adulthood. Our comprehensive care model ensures that patients receive care from physicians, hemophilia-trained nurses, social workers, physical therapists, physiatrists, and orthopedists. Care is personalized based on the latest research and advancements in treatment options.

Conditions We Treat

• Hemophilia is a group of inherited bleeding disorders in which the blood fails to clot properly, and bleeding can occur with minimal injury or trauma. People with hemophilia have low levels of clotting factor VIII (Hemophilia A), factor IX (Hemophilia B), or factor XI (Hemophilia C). Severe hemophilia A and B affect about one in every 5,000 male births, hemophilia A being far more frequent. Females are rarely affected. However, the carrier state is quite common in females, and can under certain circumstances also be associated with bleeding. Hemophilia C is generally milder and affects about one in every 100,0000 female or male births.
• Von Willebrand Disease (vWD) is a common, inherited bleeding disorder in which the blood platelets do not properly adhere to damaged or injured blood vessel wall, with common bleeding sites being mucous membranes, including the oral cavity, gums, and uterine lining. People with vWD have defective or very low levels of the Von Willebrand Factor (vWF), a clotting factor essential for normal platelet attachment to damaged blood vessel walls. The condition is found in approximately one in 1000 male or female births, making it the most common inherited bleeding disorder.
• Venous Thromboembolism, including Deep Vein Thrombosis (DVT), in which blood-flow-blocking blood clots in the deep veins of the body. These clots typically occur in the thigh, lower leg, pelvis, or arm, and Pulmonary Embolism is another category, in which part of a clot from a DVT breaks off and travels to the lungs, causing a blockage of blood flow to the lungs, chest pain, and potentially severe breathing difficulty. Venous Thromboembolisms can be associated with the use of oral estrogen.
• Platelet Function Disorders may be inherited or associated with medications.

Comprehensive Hemophilia Clinics

The Mass General Hemophilia Treatment Center (HTC) is a comprehensive pediatric and adult program that promotes caring for the whole patient including their physical, psychosocial, and mental wellbeing. We use a multidisciplinary approach to care to provide several necessary services during your visit. A comprehensive care approach has been proven to decrease medical complications and increase quality of life for patients living with bleeding disorders. Our comprehensive care team includes adult and pediatric hematologists, nurse practitioner, social workers, physical therapists, physiatrist, a clinical research coordinator and other specialists. Our goal is to provide each patient with an individualized care plan to reduce bleeding complications and hospitalizations and improve quality of life.

Specialty Clinics

In addition to the HTC, we offer several specialty clinics that may benefit our bleeding-disorder patients. After being seen our Hemophilia Clinic, we may provide referrals to one of Mass General’s many specialty clinics:

• Pediatric Stroke and Vascular Service
• Neurology/Neurosurgery
• Hereditary Hemorrhagic Telangiectasia (HHT) Center
• Pediatric Dentistry/Orthodontics

Genetic Services

• Carrier testing
• Prenatal diagnosis
• Pre-implantation genetic diagnosis
• Other services including clotting-factor and factor-inhibitor assays & DNA analysis for carrier detection

Blood Factor Program

The Massachusetts General Hospital Blood Factor Program was established as a non-profit factor home delivery service in 2014. Revenue generated through our Blood Factor Pharmacy Program has played an important role in the growth of our HTC Clinical Staff, creating more opportunities to support our patients and expand our services to our community. We have partnered with New England Life Care as our preferred Blood Factor Contract Pharmacy.

Our Blood Factor Program allows our clinical staff the ability to ship blood factor products directly to our patient’s home, conveniently and effectively. This model enables our team to more oversight into getting our patients their medication. Our team handles all eligibility determinations, prior authorizations, deliveries, blood factor management, billing, and can troubleshoot any issues patients may experience quickly.

Our Blood Factor Program is elective. Patients are free to use whichever pharmacy service they choose. Eligibility for our program is performed by our billing staff through a thorough process of determining where each patients blood factor coverage lies within their plan. All plans have both a pharmacy benefit and a medical benefit, in which we must determine where your in-home blood factor injectable coverage is found.

Services We Provide

• Home delivery
• Access to all blood factor treatments
• Expansive selection of infusion supplies
• Coverage and coordination of home infusion nursing services
• Attentive customer service
• Ongoing clarification of insurance coverage for factor waste disposal program
• Patient Education Programs
• 24 hour phone contact

Why 340B Is Important

Section 340B of the Public Health Service Act requires pharmaceutical manufacturers participating in Medicaid to sell outpatient drugs at discounted prices to health care organizations that care for many uninsured and low-income patients. One of the health care organizations or covered entities this pertains to are Hemophilia Treatment Centers. 340B generated funds allow our HTC to stretch limited federal resources to reduce the price of outpatient pharmaceuticals for patients and expand health services to the patients and communities we serve. These savings are passed on to the patient directly in terms of discounted prices of incredibly expensive medications passed on to their insurance company.

Manufacturing Co-Pay Assistance Programs

All manufacturers of blood factor products have co-pay assistance programs all patients can and should enroll in. The Co-Pay Assistance programs are tremendously beneficial as they can be utilized to cover premiums and deductibles on plans.

As we know, blood factor is tremendously expensive. January 1^st comes, and your deductible is reset. You may have $5000 deductible. If on January 2^nd you are shipped your monthly blood factor medication, your deductible will be met on that shipment. The co-pay assistance program is designed knowing the great expense of the medication, to allow you to pay your deductibles with their co-pay assistance programs.

Research and Innovation

In addition to our clinical services, we provide access to the most recent advances in education and research.

Current National Studies

American Thrombosis and Hemostasis Network (ATHN)- partners with HTCs around the country to facilitate studies to gain a better understanding of the issues affecting people with bleeding and clotting disorders.

Women and Girls with Bleeding Disorders Research studies focusing on women and girls with bleeding disorders.

Current Local Studies

The Role of the VWF Propeptide in Platelet Adhesion/aggregation and Associated Fibrin Formation in Flowing Blood from Patients with type 1 Von Willebrand Disease.  Major differences between Humate-P, a plasma-derived concentrate that contains the von Willebrand factor (VWF) and factor VIII, and Vonvendi, a recombinant VWF, include:

• The presence of factor VIII and
• An abundant amount of VWF propeptide (VWFpp), a naturally occurring additional VWF-related protein (also known as Von Willebrand Factor 2 in Humate-P but not Vonvendi)
• The presence in Vonvendi of all the VWF multimers in plasma in addition to ultra-large multimers normally found only in physiological VWF stored in the Weibel-Palade bodies of endothelial cells and in platelet -granules. The focus of this study is to determine the qualitative and quantitative effects of the VWFpp on platelet adhesion/aggregation and fibrin deposition in VWD.

Open to ages 6-65

Fitusiran Study: Fitusiran is an investigational RNA interference agent used to treat both Hemophilia A and Hemophilia B patients with and without inhibitors. Fitusiran, a once a month, subcutaneous injection, decreases antithrombin and increases thrombin (a naturally occurring clotting protein). Fitusiran is a non-factor product. Please ask your physician for further information.

Pediatric Stroke/Thrombophilia: The HTC maintains a database of patients who have thrombophilia and have experienced a thromboembolic or hemorrhagic stroke. This database is being used to investigate patterns between the presence of thrombophilia and stroke incidence.

Patient Resources and Education

• Anticoagulation Forum
• Hemophilia Alliance
• Hemophilia Federation
• Hemostasis and Thrombosis Research Society
• Hole in the Wall Gang
• National Blood Clot Alliance
• National Hemophilia Foundation
• NEHA
• Platelet Disorders Support Association (PDSA)
• Playing it Safe: Sports and Exercise
• Steps for Living
• The Foundation for Women and Girls with Blood Disorders
• Travel Resources Thrombosis and Hemostasis Societies of North America (THSNA)

Travel

If you are traveling domestically or internationally, please utilize the resources below to locate nearby HTCs:

• In the United States
• Outside the United States

Our Care Team

Meet the dedicated and passionate members of our comprehensive care team at the Massachusetts General Hospital Hemophilia and Thrombosis Treatment Center.

Eric Grabowski, MD, SCD
Pediatric Hematology Medical Director

Mariel Smith, MD
Pediatric Hematology

Larissa Bornikova, MD
Associate Medical Director
Adult Hematology

Colleen Tighe, NP
Adult & Pediatric Hematology

Nurses

Kathy Bezzarro, RN
Adult Hematology

Caroline Kriolovich, RN
Pediatric Hematology

Rhonda McIntyre, RN
Pediatric Hematology

Megan Murphy-Flood, RN
Pediatric Hematology

Patricia Scott, RN
Pediatric Hematology

Karen Sylvanowicz, RN
Pediatric Hematology

Social Work

Elyse Levin-Russman, MSW
Pediatric Hematology

Rebecca Lawrence, MSW, LICSW
Oncology Social Worker

Physiatry

Brian Wishart, DO
Pediatric Hematology

Physical Therapy

Jennifer Hathaway
Adult Hematology

Casey Vandale, PT
Pediatric Hematology

Administration

Samantha Block
Clinical Research Coordinator

Eric Cronin
Program Manager

Nurses

Caroline Kriolovich, RN
Pediatric Hematology

Rhonda McIntyre, RN
Pediatric Hematology

Megan Murphy-Flood, RN
Pediatric Hematology

Patricia Scott, RN
Pediatric Hematology

Karen Sylvanowicz, RN
Pediatric Hematology

Social Work

Elyse Levin-Russman, MSW
Pediatric Hematology

Physiatry

Dr. Brian Wishart, DO
Pediatric Hematology

Physical Therapy

Jennifer Hathaway
Adult Hematology

Casey Vandale, PT
Pediatric Hematology

Administration

Eric Cronin
Program Manager

Samantha Block
Clinical Research Coordinator