Pediatric Oncology

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.

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.

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.

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 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.

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.

The Johns Hopkins Pediatric Bone Marrow Transplant (BMT) Program is a leader in the use of haploidentical donors and the delivery of novel immune therapies for difficult-to-treat cancers, placing the BMT program at the cutting edge of cancer treatment.

Physician-scientists within pediatric blood and marrow transplantation are both devoted clinicians and researchers who strive to maximize efficacy and decrease toxicity of BMT. Our experts are working on projects in the following areas:

• Decreasing regimen-related toxicities and graft-versus-host disease (GVHD)
• Overcoming traditional limitations associated with blood stem cell donor availability and optimizing the selection of the best possible available donor.
• Developing strategies for post-transplant immunotherapy to prevent and/or treat relapse
• Understanding the scope and pace of immune recovery after using haploidentical donors and post-BMT cyclophosphamide
• Novel BMT approaches to ultra-high risk (refractory or relapsed) solid tumors
• Harnessing immune-modulatory and regenerative capacity of non-blood stem cells to reduce the development and severity of post-transplant complications like graft-versus-host disease (GVHD)

• Clinical care is delivered in the recently constructed, state-of-the-art Bloomberg Tower on the main hospital campus. The inpatient and outpatient units are located within contiguous space on the 11th floor of the Children’s Center. Our inpatient unit includes 20 spacious, single patient rooms designed specifically for our oncology and bone marrow transplant patients and is equipped with an air filtration system that allows patients to leave their rooms as they are able. In addition to a recently renovated child life room, patients have access to a large recreation room that includes a half- size basketball court.
• Our program includes seven devoted faculty members with expertise covering a broad range of bone marrow transplant indications and post-transplant complications. Our physicians are complemented by an experienced, well-trained, team of advanced practice providers, coordinators, nurses, pharmacists and social workers, along with child life and financial specialists specifically dedicated to our transplant patients. This terrific group of individuals underscores our multidisciplinary approach to patient care and family support.
• While we offer bone marrow transplant using the full spectrum of donor types and stem cell sources, our program has championed the use of haploidentical, or “half-matched,” family donors for pediatric and adolescent and young adult patients with both malignant disorders and nonmalignant conditions such as sickle cell anemia, aplastic anemia, thalassemia and a wide variety of immunologic disorders. This novel strategy has torn down traditional barriers to donor availability and essentially enables every patient in need of a transplant to successfully find a donor.
• Our established adolescent and young adult program is built upon regular interactions between pediatric and oncology faculty in a variety of areas including  bone marrow transplant, hematologic malignancies, sarcomas and brain tumors and ensures that adolescent and young adult patients have access to a broad scope of clinical trials and opportunities. The program has counselors and other experts available to address concerns such as fertility preservation and psychosocial challenges unique to this patient population. We also have an ongoing partnership with the Ulman Cancer Fund (UCF), a national organization dedicated to supporting adolescent and young adult patients and their families. The UCF is currently constructing a dedicated residence for young adults undergoing treatment in Baltimore that is in close proximity to the Children’s Center.
• We are the only center in the Mid-Atlantic to offer allogeneic bone marrow transplant using haploidentical family donors as a curative option for pediatric and adolescent and young adult patients with newly diagnosed aplastic anemia and high-risk sarcomas. Our sarcoma protocol builds upon a body of clinical data and includes the use of cutting-edge immunotherapy in the post-transplant period.