Pediatric Blood and Bone Marrow Transplant Program
Blood stem cell transplantation has a long history at Johns Hopkins. In fact, it was a Johns Hopkins Physician-scientist, George Santos, who more than 40 years ago established regimens that would lead to bone marrow transplantation as it is performed today. In 1995, we established one of the first pediatric bone marrow transplant programs in the country. Today, the program at Johns Hopkins is inter-nationally recognized and provides outstanding care to pediatric and young adult patients in need of a blood stem cell transplant procedure. In 2016, under the leadership of Drs. Small, Cooke and Cohen, The Johns Hopkins Division of Pediatric Oncology and BMT Ranked #5 Best Hospital for Pediatric Cancer by US News and World Reports. Physicians at the Johns Hopkins Kimmel Cancer Center and the Johns Hopkins Children’s Center work together on a number of transplant options or protocols – many developed here at Hopkins-- that are curing children, adolescents and young adults with a variety of cancers and other disorders of the blood, bone marrow and immune system. Our program is a leader in clinical care and research initiatives and is committed to raising the standard of care for BMT recipients.
The Pediatric BMT program at Hopkins is recognized as a Children’s Oncology Group (COG) designated transplant center. A unique aspect of the program is its seamless integration into the larger, nationally recognized adult bone marrow transplant program at the Johns Hopkins Kimmel Cancer Center headed by Dr. Richard Jones. The combined program has Foundation for the Accreditation of Cellular Therapy re-accreditation and shares the following: a common stem cell processing facility, meetings to discuss relevant patient issues, clinical protocols, and resources for data collection and monitoring.
To date, more than 5,000 bone marrow transplants have been performed at the Johns Hopkins Kimmel Cancer Center, a National Cancer Institute-designated Comprehensive Cancer Center that is fully accredited by the National Marrow Donor Program as an unrelated donor transplant center. As a national referral center for bone marrow transplant, Kimmel Cancer Center experts perform around 300 (combined pediatric and adult) transplants each year.
Bone Marrow Transplant Program
The Pediatric Bone Marrow Transplant Program is directed by Dr. Kenneth Cooke, an internationally known bone marrow transplant physician recognized as an expert in transplant related complications and developing multicenter, translational research protocols. Since his arrival, Dr. Cooke has been influential in the development of the bone marrow transplant program at the Kimmel Cancer Center, having identified and accomplished many initiatives to promote its growth both internally and around the world.
Our program provides transplantation of hematopoietic (or blood) stem cells that are derived from the bone marrow as a treatment option for a variety of acquired and congenital disorders of children and young adults. These include: acute and chronic leukemia, Hodgkin and non-Hodgkin lymphomas, sarcomas, brain tumors, bone marrow failure syndromes, inherited immune deficiency syndromes and metabolic disorders. The program has significant experience using both myeloablative and reduced intensity conditioning regimens for allogeneic transplants, followed by the delivery of blood stem cells collected from the bone marrow, placenta and peripheral blood of fully and partially-matched related and unrelated donors.
As another novel approach to treatment, high dose chemotherapy with autologous stem cell rescue is available as a tandem and non-tandem option for many solid tumors as is high dose cyclophosphamide (Cytoxan) therapy alone for select bone marrow failure and autoimmune syndromes.
Taking care of transplant patients is one of the most complicated and yet rewarding areas of medicine. “Bone marrow transplant isn’t just a procedure – it’s a platform from which other forms of cancer therapy can be given,” says Dr. Cooke. Moreover, “Bone marrow transplant represents an ongoing approach to care. The procedure itself is rather straightforward, but the provision of post-transplant care in the days, weeks, months and years that follow that absolutely require a dedicated, well-trained group of practitioners.” The pediatric bone marrow transplant team at the Kimmel Cancer Center combines years of patient care experience, expertise in supportive care, transplant-related complications and survivorship, with direct links to national cooperative groups.
Transplant patients are managed by an integrated team made up of specialized physicians, nurse practitioners, physician assistants, pharmacists, social workers, and registered nurses. In keeping with our multi-disciplinary approach to clinical care, patients with non-malignant disorders are co-managed by members of the BMT program and the pediatric subspecialty area with expertise in the disease being treated (e.g. immunology, hematology, endocrinology, genetics and neurology). We also have families of children with a similar diagnosis available to serve as one-on-one support for current patients.
Inpatient care is delivered at the state-of-the-art Bloomberg Children’s Center in a 20-bed, HEPA-filtered unit dedicated solely to the care of oncology and bone marrow transplant patients. Outpatient care is provided in an equally advanced clinic connected to the inpatient unit.
Our mission is simple: to make sure that every patient in need of a bone marrow transplant has a suitable donor, and to minimize the toxicity and maximize the efficacy of our transplant protocols.
Haploidentical or “Half-matched” Blood Stem Cell Transplantation
Historically, children in need of a blood stem cell transplant required a human leukocyte antigen (HLA) identical or “fully matched” bone marrow donor. The best matched donor is usually a sibling, but only about 25% of children in need of a transplant will have a “fully matched” sibling donor. In addition, only approximately 30% of patients can find a perfectly matched unrelated donor in the national bone marrow and cord blood registries; and this percentage is much lower for minority groups, especially African Americans and Latino Americans.
At the Kimmel Cancer Center, we developed a unique method of using “half-matched” donors to ensure that every patient in need of a bone marrow transplant has a donor that can be rapidly identified. A child inherits half of their bone marrow from their mother and half from their father, meaning in almost every situation a parent can be the bone marrow donor, and 50% of siblings are half-matched too. This dramatically increases the number of patients who can successfully be transplanted. In haploidentical BMT, parents, siblings, and potentially aunts and uncles, nieces and nephews, half-siblings, and grandparents can safely serve as donors. Our experts have performed more than 500 haploidentical transplants for adult and pediatric patients with safety and toxicity comparable to traditional transplants. This safety has made it possible for haploidentical stem cell transplantation to be used to treat many types of cancerous and non-cancerous pediatric diseases.
Care for Adolescent and young adults with Cancer and other blood disorders
The pediatric oncology and bone marrow transplant programs have a keen focus on the unique medical and psycho-social challenges that face the adolescent and young adult population. At Johns Hopkins, young adults can be admitted and cared for in the Bloomberg Children’s Center up to age 25. The programs are also associated with the Ulman Cancer Fund, an internationally recognized organization dedicated to supporting adolescent and young adult patients with cancer. The headquarters is in Baltimore, and the Kimmel Cancer Center’s pediatric oncology program is one of the few programs in the country that have a Ulman Cancer Fund-sponsored patient navigator to assist with care delivery.
Bone Marrow Transplant for non-malignant disorders
The Pediatric Bone Marrow Transplant program is also using blood stem cell transplantation from suitably matched donors to treat patients with non-malignant disorders of the blood, immune and metabolic systems. When possible, reduced intensity bone marrow transplant preparative regimens are used to minimize both short term and long term side effects from chemotherapy and radiation therapy. When a “fully matched” donor is identified, we have protocols to minimize the amount and duration of immunosuppressive drugs that are needed after bone marrow transplant. This approach allows patients to come off of their medication earlier and helps the immune system recover faster.
Non-malignant disorders we are currently treating include: Severe Combined Immunodeficiency, Chronic Granulomatous Disease, Hyper-IgM syndrome, Common Variable Immunodeficiency, Hemophagocytic Lymphohistiocytosis, Bare Lymphocyte syndrome types I and II, Severe Aplastic Anemia, Fanconi anemia, Dyskeratosis Congenita, Diamond-Blackfan Anemia, Hunter syndrome, Hurler syndrome, Adrenoleukodystrophy, Metachromatic Leukodystrophy, Sickle Cell Disease, and Thalassemia.
The Hopkins BMT program has done pioneering work using haplo-identical donors and reduced intensity conditioning to cure many patients with Sickle Cell Disease and we are currently using this platform to treat pediatric patients with Sickle Cell Disease and Thalassemia throughout the world.
Translational Research Efforts
What sets the Kimmel Cancer Center’s Pediatric Bone Marrow Transplant program apart from other children’s hospitals is its research mission centered on improving ways to provide the best possible care to our patients. The Kimmel Cancer Center is a leader in the use of haplo-identical donors and the delivery of novel immune therapies for difficult to treat cancers, placing the Bone Marrow Transplant program at the cutting edge of cancer treatment. “We have a venue at the Kimmel Cancer Center to deliver novel, immune cell-based, therapies to our patients, we will be driving the next generation of therapies to bring transplant to a large population of patients,” says Dr. Cooke. “The only way to break new ground is through well planned, carefully controlled translational research, and the Johns Hopkins Kimmel Cancer Center provides a terrific environment for this to be successful. Physician-scientists within Pediatric Blood and Marrow Transplantation are both devoted clinicians and researchers who strive to maximize efficacy and decrease toxicity of bone marrow transplant.
Clinical Research Protocols
The Pediatric Bone Marrow Transplant program currently has several open clinical trials for patients with Leukemia, Lymphoma, High risk solid tumors, Sickle Cell Disease, Beta Thalassemia, severe aplastic anemia and Immune deficiencies.
To make an appointment, please call our referral coordinator at 443-287-6997. International patients please contact International Intake at 410-502-7683.
Physicians calling after hours or on weekends may call the Hopkins Access Line (HAL) 24 hours a day, at 410-955-9444 or 1-800-765-5447; ask for the pediatric oncology attending physician.
Kenneth Cooke, MD: Dr. Ken Cooke joined the faculty of the Sidney Kimmel Cancer Center in 2013. He was recruited as the Herman and Walter Samuelson Professor in Oncology to serve as the overall Director of Pediatric BMT Program. He is internationally known for his expertise in stem cell transplantation and the two most frequent and severe complications following this procedure: graft-versus-host disease (GVHD), and non-infectious lung dysfunction that occur both early and late after BMT. He has brought laboratory insights back to the bedside in the form of several clinical trials to treat GVHD and lung dysfunction after BMT. His work in this area resulted in his induction into the American Society of Clinical Investigation.
Heather Symons, MD: Dr. Heather Symons is the clinical director of the Pediatric BMT program. Dr. Symons’ research focuses on the development of novel immunotherapies for hematologic and high-risk solid tumors and on overcoming traditional limitations to donor selection. Her focus is on using haplo-identical donors for blood stem cell transplantation and she is currently working toward strategies to identify and select the best half-matched donor available.
Allen Chen, MD: Dr. Chen is the senior member of the BMT teams and served as the director of the program until 2013. Dr. Chen oversees quality improvement efforts in the Cancer Center. His clinical interests are in metastatic neuroblastoma, lymphoma, and developing reduced intensity conditioning regimens for patients with inherited diseases of the marrow and immune system.
Christopher Gamper, MD PhD: Dr. Gamper’s research focuses on how to train the newly engrafted donor immune system to efficiently target and destroy infected tissues and tumor cells in the BMT recipient without causing graft-vs-host disease. From a clinical perspective, Dr. Gamper is developing clinical trials to identify novel approaches to treat acute GVHD that fails to respond to steroids. He is also spear heading efforts to develop safer “reduced intensity” protocols for patients with sickle cell disease and thalassemia using half-matched parents or siblings as donors, based on the safety and success of such an approach developed by colleagues in the adult BMT program at Johns Hopkins.
Elias Zambidis, MD PhD: Dr. Zambidis is an eminent scholar with a research interest in advancing out understanding of pluripotent stem cells—‘master’ stem cells that can generate any new cell or tissue that the body needs to repair itself. His lab (The Zambidis Laboratory) at the Johns Hopkins Institute for Cell Engineering continues to find new ways to use special reprogrammed stem cells to treat severe blood-related and vascular diseases. Dr. Zambidis’s clinical interests focus on patients with immune deficiencies or immune dysregulatory diseases.
Nicolas J Llosa, MD: Dr. Llosa recently joined the program after completing his fellowship training at Hopkins. His research focuses on understanding how the immune system interacts with cancer cells from sarcoma tumors. Immunotherapy is a type of cancer treatment designed to boost the body's natural defenses to fight the cancer and it is currently one of the most promising approaches for treating some types of solid tumors. His specific area of interest is the use of drugs that target immune system checkpoints.
Orly Klein, MD: Dr. Klein recently joined the program after completing her fellowship training at Hopkins. Her research focuses on how inflammation that is generated after BMT can injure vascular endothelial cells – or blood vessel lining cells - and result in lung injury and dysfunction early after transplant.
Other Team Members
Nancy Robey, PA: Advanced practice provider with expertise in bone marrow transplant
Mary Jo Holuba, CRNP: Advanced practice provider with expertise in bone marrow transplant
Nicole Dores, PharmD: Bone marrow transplant specialty pharmacist
Christine Manuel-McGuiggin: Bone marrow transplant case manager / coordinator.
Michelle Kokoszka, RN: Discharge coordinator
Patrice McMullen, RN: Bone marrow transplant Insurance specialist
Stacey Starling: Bone marrow transplant insurance specialist
Megan Burris, RN: Unite Arab Emerates Clinical Coordinator
Mohammed Al Taee: International Care Coordinator
Sahar Ismael: International Care Coordinator
Khalid Mohamed Ali, MD: International Care Coordinator
Mustafa Hamid: International Care Coordinator
Learn More About the Pediatric Blood and Bone Marrow Transplant Program
Pediatric Blood and Bone Marrow Transplantation | FAQ with Dr. Kenneth Cooke