Overview:

The Childhood and Adolescent Bone Marrow Failure Program at the Maria Fareri Children’s Hospital at Westchester Medical Center and New York Medical College 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 is involved 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)
  • Others

 

Rare genetic and metabolic disorders:

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

 

 

Key Team Members/Disciplines:

  • Director: Cori Abikoff, MD
  • Stem Cell Transplantation: Mitchell S. Cairo, MD
  • Stem Cell Transplantation Coordinator: Tamar Horowitz, RN, MSN, CPNP, CPHON
  • Genetics: David Kronn, MD
  • Radiation: Chitti Ramanamoorthy, MD
  • Stem Cell Processing: Janet Ayello, MS
  • Hematopathology: Humayun Islam, MD
  • Pediatric Cardiology: Deborah Freidman, MD
  • Pediatric Immunology: Subhadra Siegel, MD
  • Pediatric Orthopedics: Samuel Kenan, MD
  • Clinical Research Nurse Coordinator: Erin Morris, RN
  • Biostatistics: Qiuhu Shi, PhD
  • Social Work: Rose Bartone, LCSW-R and Barbara McLain, LCSW
  • Clinical Pharmacist: Liana Klejmont, PharmD

 

Unique Features:

  • Comprehensive Genetic Diagnostic Program
  • Genetic Consulting
  • Targeted Immunotherapy
  • Novel Therapeutics
  • Blood and Marrow Transplantation
  • Unrelated Cord Blood Stem Cell Transplantation
  • Unrelated Adult Donor Stem Cell Transplantation
  • Haploidentical Stem Cell Transplantation
  • T-cell Depletion and Stem Cell Selection Transplantation
  • Reduced Intensity Conditioning Therapy
  • Hepa filtered and Positive Pressure Protected Isolation Room
  • Cellular and Tissue Engineering Laboratory
  • Multidisciplinary Team
  • Donor Lymphocyte Infusions

 

Research and Education Highlights:

  • ACGME accredited Pediatric Hematology, Oncology and Stem Cell Transplantation Fellowship Program
  • Student and Resident elective/selectives
  • FACT Accredited
  • NMDP Accredited
  • Reduced Intensity Conditioning Program
  • Long Term Survivors Program
  • Bone Marrow Failure Genetics Diagnostics
  • Novel Therapeutics