Programs

Programs

Programs We Offer

Maria Fareri Children’s Hospital provides an unmatched level of care in the region–drawing upon both standard protocols and novel therapies to tailor care to each patient and advance the state of medicine itself. Driven by research and delivered to patients, our hematology and oncology programs represent the gold standard of preventative, diagnostic, innovative and therapeutic strategies. With specialists available for consult around the clock, our Children and Adolescent Cancer & Blood Disease Center provides state-of-the-art, compassionate, and customized care for each young child and adolescent in the following pediatric clinical and therapeutic programs within this center:

Hematological Malignancies

Hematological Malignancy (Leukemia/Lymphoma) consist of a multidisciplinary team focused on the prevention, diagnosis, and treatment of cancers arising in the bone marrow, lymph nodes and other blood-developing organs. The cross-disciplinary team consisting of pediatric oncologists, surgeons, pathologists, radiation oncologists, bone marrow transplantation, psychology, neuropsychology and social workers. 


Histiocytic Disorders

The histiocytosis program consists of a multidisciplinary team focused on the diagnosis and treatment of histiocytic disorders in children and adolescents. Histiocytic disorders are a group of diseases that occur when there is an overproduction of histiocytes or mononuclear phagocytic cells of bone marrow origin. Overproduction of these cells can lead to organ damage and cause disease, based on the type of the cells involved. Correct and complete pathologic diagnosis is essential because the different diseases require different treatment approaches. We focus on dendritic cell disorders, including Langerhans Cell Histiocytosis (LCH), Juvenile Xanthogranuloma (JXG) and Erdheim Chester Disease (ECD); macrophage cell disorders, including Hemophagocytic Lymphohistiocytosis (HLH) and Rosai-Dorfman Disease (RD); and malignant histiocytosis, including certain kinds of leukemia and malignant tumors. 


Neuro-Oncology

Our neuro-oncology program provides both standard treatment plans and research protocols, as well as individualized therapy driven by novel cellular and immune modalities. The crossdisciplinary team includes pediatric neurosurgery, pediatric neuro-oncology, pediatric radiation oncology, pediatric neuropsychology, and pediatric neurology. 


Solid Tumors

The solid tumor program focuses on solid tumors, abnormal masses of tissue, in children, adolescents and young adults, including neuroblastoma, kidney tumors such as Wilms’ tumor, liver tumors such as hepatoblastoma and hepatocellular carcinoma, bone tumors such as Ewing’s sarcoma and osteosarcoma, soft tissue tumors such as rhabdomyosarcoma, and germ cell tumors in the ovaries and testicles. The multidisciplinary team includes pediatric oncologists, surgeons, urologists, pathologists, radiation oncologists, gynecologists, otolaryngologists and social workers. 


Bone Marrow Failure

The Childhood and Adolescent Bone Marrow Failure Program consists of a multidisciplinary team focused on the diagnosis, etiology, biology and treatment of children and adolescents with a variety of bone marrow failure disorders. Several state-of-the-art programs include Genetic Diagnosis, Immunotherapy and Blood and Marrow Transplantation. The program is dedicated to the training and mentoring of residents and fellows who are interested in pursuing clinical, translational and/or basic science research in bone marrow failure syndromes. The bone marrow failure program also specializes in the treatment of rare genetic and metabolic conditions, which can be cured with bone marrow transplantation and are included in clinical research to advance the field of transplantation for these disorders. Specifically, the program is designed to provide personalized and customized therapy for each child and adolescent who is diagnosed with a bone marrow failure syndrome or rare genetic disorder. 

Diseases 
This comprehensive program is focused on all diagnostic, genetic and therapeutic aspects of: 

Bone marrow failure syndromes: 

• Amegakaryocytic Thrombocytopenia
• Aplastic Anemia
• Congenital Neutropenia (Kostmann syndrome)
• Diamond Blackfan (Red Cell Aplasia) Anemia
• Dyskeratosis Congenita
• Fanconi Anemia
• Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Reticular Dysgenesis
• Schwachman-Diamond Syndrome
• Thrombocytopenia with Absent Radii (TAR) 

Rare genetic and metabolic disorders: 

• Adrenoleukodystrophy
• Diseases of IL10 and IL10 Receptor
• Hurler Syndrome
• Krabbe Disease
• Metachromatic Leukodystrophy
• Niemann Pick Disease
• Osteropetrosis 


Primary Immunodeficiencies

The Childhood Primary Immunodeficiency Program is a multidisciplinary and comprehensive program dedicated to the diagnosis, prevention, treatment and long term follow up of children diagnosed with primary immunodeficiency syndromes. The program consists of several state-ofthe- art programs in Newborn Screening, Prevention, Genetic Diagnosis, Stem Cell Transplantation, Reduced Intensity Conditioning and Adoptive Cellular Immunotherapy. The program also provides a unique opportunity for clinical, translational and basic research for students, residents and fellows focused in the biology and treatment of primary immunodeficiency in children. Most importantly, the program is designed to provide customized and personal care and long-term follow-up to each child with a primary immunodeficiency. 

Diseases 
The Primary Immunodeficiency Program is focused on all known immunodeficiencies including: 

• Ataxia-Telangiectasia
• Autoimmune Neutropenia
• Complement Deficiencies
• Humoral Immunity
• Agammaglobulinemia (Bruton’s)
• Common Variable Immunodeficiency
• Hyper IgE Syndrome
• Hyper IgM Syndrome
• IgA Deficiencies
• IgM Deficiency
• Severe Combined Immune Deficiency (SCID)
• Neutropenia
• Congenital Neutropenia
• Cyclic Neutropenia
• Neonatal Neutropenia
• Reticular Dysgenesis
• Wiskott-Aldrich Syndrome
• Phagocyte Function
• Chediak-Higashi Syndrome (CHS)
• Chronic Granulomatous Disease (CGD)
• Leukocyte Adhesion Deficiency
• Specific Granule Deficiency 


Sickle Cell Disease

The sickle-cell disease program unites an experienced team of pediatric hematologists, bone marrow transplant specialists, neuropsychologists, nurses, and social workers advancing both standard and novel treatment strategies. Identified through the Hudson Valley Newborn Screening Program, patients and their families receive care in a family-centered atmosphere, with testing and appointments with sub-specialists accomplished on the same day. This program is the founding center in the Hapliodentical Sickle Cell Disease Consortium stem cell transplant clinical trial, a study that recruits parents as bone marrow donors to replace sickle red blood cells with healthy red blood cells. 


Thrombosis and Hemostasis

Our team is dedicated to treat children with bleeding and clotting problems. The team includes physicians, nurses and social workers, with special interest in hemophilia and coagulation factor deficiencies. We also dedicate our care to children with clotting disorders. Beyond the care of children afflicted with these conditions, we also consider family education, social interactions among families of affected children, home care and support as the goal of our team to help parents and families with their inevitable emotional challenges. We engage in ongoing research for developing innovative therapies and for addressing safety issues, such as complications or risks that may be associated with medications. We educate and mentor future trainees in diagnosis and treatment of children and adolescents with bleeding and clotting disorders, and treatment. 

Diseases 
Our team is dedicated to care for children with bleeding problems. We treat children with:

• Congenital or acquired bleeding disorders:
• Hemophilia A (Classical Hemophilia-factor VIII deficiency)
• Hemophilia B (Christmas disease, factor IX deficiency)
• Hemophilia C (Factor XI deficiency)
• Von Willebrand’s disease
• Rare clotting factor deficiencies:
• Factor I (fibrinogen deficiency, known as afibrinogenemia or dysfibrinogenemia/hypofibrinogenemia)
• Factor II (prothrombin)
• Factor V (parahemophilia)
• Factor VII
• Factor X (Stuart factor)
• Factor XII (Hageman factor)
• Factor XIII
• Disorders of the platelets:
• Hermansky-Pudlak syndrome
• Wiskott Aldrich syndrome
• Gray platelet syndrome
• Alpha granule deficiency
• Bernard Soulier Syndrome or disorders involving fibrinolytic system (e.g. PAI-I deficiency or alpha-2 antiplasmin deficiency)
• Inherited Disorders of Thrombosis:
• Protein C
• Protein S
• Anti-thrombin-III
• Factor V Leiden
• Prothrombin gene mutation
• Anti-phospholipid antibodies
• Lipoprotein-A
• Homocysteinemia
• Elevated fibrinogen
• Factors VIII, IX, XI
• Low factor XII

Blood and Marrow Transplantation

Found in adult bone marrow and a baby’s umbilical cord, stem cells carry the astonishing potential to develop into different cell types, creating an internal repair network that replenishes cells without limit. For a baby or child with cancer or blood disease, an influx of healthy stem cells can restore the ability to produce healthy blood and immune cells, setting the patient on a course toward wellness. Our Blood and Marrow Transplantation (BMT) Program consists of a multidisciplinary team of specialists dedicated to the therapeutic use of bone marrow, peripheral blood (stem cells released from marrow to bloodstream), cord blood and cellular therapy for children, adolescents, and young adults with malignant, non-malignant and serious blood-related disorders. 


Developmental and Experimental Therapeutics

The Developmental and Experimental Therapeutics program focuses on translational and clinical application of new therapeutic strategies in early-phase clinical investigation. This program is designed to gauge the safety, feasibility, dose, schedule and early efficacy of new therapeutic strategies during experimental clinical investigation in children, adolescents, and young adults, with both malignant and non-malignant blood disease-related disorders. 

Our areas of expertise include:

• Phase I/II Therapy
• Bench to Bedside Therapy (Translational)
• Humoral Immunotherapy
• Radioimmunotherapy
• Cellular immunotherapy
• Stem Cell Transplantation
• Targeted Cellular Therapy
• Immunotoxin Therapy 

These specialties yield the opportunity for students, residents, and fellows to pursue scholarly research in this field. Most importantly, this program provides personal and customized medical therapy and care to children, adolescents, and young adults. 


Hematotherapy

Clinical services don’t have to feel so…clinical. Our full-service apheresis and transfusion center provides care to patients of all ages, including children at Maria Fareri Children’s Hospital. The newly-renovated, kid-friendly outpatient center has individual patient bays for comfort and privacy, flat-screen televisions and complimentary Wi-Fi. We also provide comprehensive inpatient services. 

We offer consultation and treatment plans for many types of apheresis including:

• Therapeutic plasma exchange
• Peripheral stem cell collection
• Red cell exchange
• Extracorporeal photopheresis
• Leukapheresis and plateletpheresis
• Blood transfusions 

The Hematotherapy Center has equipment to transfuse blood components and infuse blood product derivatives and medications, such as:

• Red blood cell or platelet infusions for outpatients with cancer and other diseases
• Factors, iron and other infusion treatments
• Therapeutic phlebotomy for conditions such as hemochromatosis or polycythemia vera 

As with all clinical services, physicians are available 24 hours a day.