Mount Sinai has a long history of excellence in hematology in all the areas of clinical care and research. Physicians, nurse practitioners, social workers, and other members of the health care team who specialize in both adult and pediatric hematology work together to assure that all patients have access to comprehensive, state-of-the art care.
An active laboratory research program seeks to provide more effective therapy for a variety of hematological conditions. In addition to three programs in hemophilia, sickle cell disease, and thrombophilia, our physicians provide care to children with anemia secondary to hemoglobinopathy, red cell membrane or enzyme disorders, aplastic anemia (including Fanconi's anemia), thrombocytopenia (ITP), neutropenia, polycythemia, and thrombocytosis.
Our treatment programs include:
The Comprehensive Hemophilia Treatment Program
Children with hemophilia and other congenital bleeding disorders are seen in the Pediatric Hematology Clinic with the guidance and leadership from the Hemophilia Center. Our comprehensive approach to patients with chronic bleeding disorders has been shown to greatly improve the outcome and the quality of life for these children. Mount Sinai was one of the first medical centers in the country to establish a multidisciplinary treatment center staffed by dedicated individuals with specific expertise and experience in the care of children and adults with hemophilia. The Mount Sinai Regional Hemophilia Treatment Center is part of a nation-wide network of federally funded hemophilia centers. Mount Sinai is the Regional Grantee for the New York metropolitan region. The reputation of Mount Sinai goes far beyond New York and the United States especially with the quality of the musculoskeletal program under the leadership of Marvin Gilbert, MD.
Christopher Walsh, MD, PhD, a noted expert in gene therapy for Hemophilia and other blood diseases, leads the Hemophilia Program. Basic research conducted at Mount Sinai is leading to improved treatments. Our staff includes nurses, social workers, a clinical coordinator, a physical therapist, a geneticist, and infectious disease and psychiatrist consultants exclusively dedicated to the patients in the hemophilia center. Education plays a critical role in the care of the patients. Integration in "normal" life is one of the goals of the program including teaching home care and identifying appropriate physical activities. Patients have an opportunity to participate in clinical trials involving the use of safer blood products, new approaches to treatment, and surveillance for new possible infections. Pediatric patients participate in camp, field trips and entertaining and social activities. Nurses and social workers visit the home and school to educate the families, teachers and classmates involved in the care of the children.
The Thrombophilia Program
Thrombosis, or blood clots, has become a "hot" issue in pediatric hematology mainly because the recognition of genetic predispositions to the development of blood clots and the increased use of central venous (IV) lines to facilitate therapy in children with a variety of conditions. Congenital predisposition for thrombosis, however, has been shown to be a significant risk factor not only for central line associated thrombosis but also for spontaneous thrombosis. Identification of young women at risk for blood clots because of the use of oral contraceptives or pregnancy has become an issue in adolescent medicine. Care of children and adolescents with thrombosis and stroke is an evolving area that requires specific expertise. The Thrombophilia Program at Mount Sinai provides the latest tests to identify the cause of blood clots and uses the most effective medications to treat existing clots and prevent the formation of new ones.
The Sickle Cell Program
Our Pediatric Hematologists have been delivering state-of-the-art care to all patients with Sickle Cell Disease for several decades. Basic research at Mount Sinai has included gene therapy for hemoglobinopathy, studies on cell interactions that lead to the excessive adhesion of red cells and enhancement of fetal hemoglobin to minimize the symptoms of sickle cell disease. Our Sickle Cell Program is a comprehensive program designed to provide holistic care. Patients have access to social workers, genetic counselors, a dedicated cardiologist, pulmonologists, neurologists, and psychologists. Together with our experienced Pediatric Intensive Care Unit staff we treat the most life threatening complications such as stroke and acute chest syndrome. The blood bank provides accessibility to hemopheresis for emergencies. The philosophy of care calls for close collaboration with the pediatrician who is providing "well child care" to the patient. Visits to the Sickle Cell Clinic emphasize care of the sickle cell disease related complications and education. Children and their families have opportunities to participate in camp and other extracurricular activities. Parents and children learn how to access information about their disease through the library, the web, and group discussion.
Young patients with various forms of cancer are treated by the Division of Pediatric Hematology/Oncology. Through membership in the Children's Oncology Group (COG) some patients have the option of enrolling in cutting-edge treatment studies.
Great progress has been made in the development of successful treatment programs for children and adolescents with cancer. These improvements have been possible because of the availability of pediatric cancer treatment centers with collective expertise in the clinical management of children with cancer and the existence of a network of experienced investigators and allied health professionals who recognize the central importance of randomized clinical trials as the best available method for identifying more successful treatment strategies and who have the resources to evaluate new treatment modalities as they become available.
The importance of comprehensive, multidisciplinary treatment in improving patient outcome has been well documented for children with acute lymphoblastic leukemia, non–Hodgkin lymphoma, brain tumors, rhabdomyosarcoma, Wilms' tumor, and Ewing sarcoma. Almost 80% of these children can be treated successfully if modern diagnostic and therapeutic approaches are initiated expeditiously. Early detection, accurate diagnosis, and appropriate treatment depend on a multidisciplinary treatment approach to children and adolescents with cancer, an approach that is uniquely available at a pediatric cancer center. The roles of specialized nursing, pharmacy, rehabilitation, and paramedical personnel and access to increasingly complex equipment and facilities are critical to improving long-term survival and quality of life.
Hematopoietic Cell Transplant Service
Hematopoietic Cell Transplants (HCT) are used to treat malignant (cancer) and non-malignant (not cancer) illnesses. HCT is a treatment that involves high-dose chemotherapy and/or powerful immunosuppressants, oftentimes in conjunction with radiation therapy. These treatment modalities are necessary in order to permit the body's acceptance of the new hematopoietic stem cells, which are immature blood cells. Additionally, these treatments rid the body of the diseased bone marrow and, in the case of cancer, cancer cells. The hematopoietic stem cells are then given to the patient in order to "rescue" the patient's bone marrow.
The type of transplant to be performed depends on several factors, including the appropriateness of identified donors/donor units and the patient's disease. The cells from the donor should match the patient's. Cell matching or compatibility is evaluated through a process called HLA-typing, which requires a blood test. HLA stands for human leukocyte antigens: these antigens are proteins found in the surface of white blood cells (leukocytes).
An autologous transplant uses the patient's own stem cells: Certain diseases do not affect the bone marrow, so the patient's own hematopoietic cells are healthy and can be used for the transplant after the body is exposed to high-doses of chemotherapy that may permanently destroy the marrow.
An allogeneic transplant uses progenitor cells collected from another person, either unrelated or unrelated to the patient. The donor may be a sibling, an unrelated adult volunteer donor identified through a donor registry, or umbilical cord blood from a relative or unrelated donor. The higher the degree of HLA-matching between donor and patient, the higher the likelihood of engraftment (acceptance of the donor cells by the patient). In the case of umbilical cord blood transplants, the cell dose (number of cells in a banked cord blood unit) is as important as the degree of HLA-matching. Most people need an allogeneic transplant because either cancer cells have invaded their bone marrow or a disease has caused the bone marrow to fail to work properly.
The Mount Sinai Division of Pediatric Hematology/Oncology offers hematopoietic cell transplantation (HCT) services that include bone marrow transplantation, peripheral blood stem cell transplantation and umbilical cord blood transplantation. Autologous (self) and allogeneic (non-self) transplants are offered; allogeneic donors can be the patient's relatives, volunteer-unrelated donors, or umbilical cord blood units from relatives or unrelated donors.
Our pediatric transplantation service is part of the Mount Sinai Adult Bone Marrow Transplantation Program, directed by Luis Isola, MD. We are a member of the National Marrow Donor Program and is approved by the AABB (formerly American Association of Blood Banks) and the Foundation for the Accreditation of Cellular Therapy (FACT). The Mount Sinai Adult Bone Marrow Transplantation Program transplants approximately adult 100 patients per year and uses a laminated air flow (LAF) unit for the highest-risk transplants. Children and adolescents that undergo an allogeneic transplant are admitted to this LAF unit.
We work in partnership with the Mount Sinai Transfusion Medicine Service, whose stem cell laboratory is accredited to perform CD34 selection.
In addition to being FACT-accredited, we are a Children's Oncology Group (COG) — approved Blood and Marrow Transplantation Center, as well as a member of the Pediatric Blood and Marrow Transplant Consortium.
Pediatric patients that require HCT are offered standard-of-care transplant regimens as well as the opportunity to enroll in transplant clinical trials through Mount Sinai as well as well as through COG.
We are one of three centers in the nation that has opened a clinical research study for hematopoetic cell transplant of inborn errors of metabolism. The study (UCPT-002) is sponsored by Aldagen. Diagnoses that can be considered for transplant on this study include the following:
- Hurler Syndrome
- Hurler-Scheie Syndrome
- Hunter Syndrome (MPS II)
- Sanfilippo Syndrome (MPS III)
- Maroteau Lamy Syndrome (MPS VI)
- Krabbe Disease (Globoid Leukodystrophy)
- Metachromatic Leukodystrophy (MLD)
- Adrenoleukodystrophy (ALD and AMN)
- Sandhoff Disease
- Tay Sachs Disease
- Neiman-Pick Disease (A, B, AB)
In addition, there is an ongoing research group consisting of physicians, medical students and a social worker dedicating their efforts to the study of in vitro fertilization with therapeutic intent, particularly for patients with sickle-cell disease.
Diseases that we consider for transplantation include, but
are not limited to:
- Acute Lymphoblastic Leukemia
- Acute Myeloid Leukemia
- Myelodysplastic Syndrome
- Wilms' Tumor
- Non-Hodgkin's Lymphoma
- Central Nervous System Tumors
- Chronic Myeloid Leukemia
- Congenital Amegakaryocytic Thrombocytopenia
- Desmoplastic Small Cell Tumors
- Germ Cell Tumors
- Hemophagocytic Lymphohistiocytosis
- Hodgkin's Disease
- Hurler Syndrome
- Severe Combined Immunodeficiency Syndrome
- Severe Congenital Neutropenia
- Sickle Cell Disease
- Wiskott-Aldrich Syndrome
- Aplastic Anemia
- Fanconi Anemia
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