At Memorial Sloan Kettering Cancer Center we have expertise in and treat children with the following conditions:

Fanconi Anemia and Other Inherited Bone Marrow Failure Syndromes (BMFS)

• Fanconi Anemia
• Congenital Amegakaryocytic Thrombocytopenia (CAMT)
• Diamond-Blackfan Anemia (DBA)
• Dyskeratosis Congenita
• GATA2 Deficiency
• Shwachman- Diamond Syndrome
• Thrombocytopenia-Absent Radii (TAR) Syndrome

Acquired Bone Marrow Failure Syndromes (BMFS)

• Aplastic Anemia
• Myelodysplastic Syndrome (MDS)
• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Sickle Cell Disease, Thalassemia, and Other Inherited Hemoglobin Disorders

• Sickle Cell Disease (SCD) / Sickle Cell Anemia
• Transfusion-Dependent Thalassemia / Transfusion-Dependent β-Thalassemia (TDT)
• Other Hemoglobinopathies

Primary Immunodeficiencies (PIDS), Primary Immune Regulatory Disorders (PIRDs) and White Blood Cell Disorders

White Blood Cell Disorders

• Chronic Benign Neutropenia (CBN)
• Chronic Granulomatous Disease (CGD)
• Cyclic Neutropenia
• Leukocyte Adhesion Deficiency (LAD)
• Severe Congenital Neutropenia (SCN) (Kostmann Syndrome or Severe Chronic Neutropenia)

Other Primary Immunodeficiencies

• CD40 ligand (CD40L) Deficiency (Hyper IgM syndrome)
• Common Variable Immunodeficiency (CVID) / Combined Immunodeficiency (CID)
• Myeloperoxidase Deficiency (MPO Deficiency)
• Severe Combined Immunodeficiency (SCID)
• Wiskott-Aldrich Syndrome

Immune Regulatory Disorders

• Autoimmune Lymphoproliferative Syndrome (ALPS)
• Chediak-Higashi Ssyndrome
• Hemophagocytic Lymphohistiocytosis (HLH)
• Immunodysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome
• Very Early-Onset Inflammatory Bowel Disease (IBD)
• X-linked inhibitor of apoptosis protein (XIAP) deficiency

Autoimmune Blood Cell Disorders

• Evans Syndrome
• Refractory Immune Thrombocytopenia (ITP)
• Refractory Autoimmune Hemolytic Anemia (AIHA)

Inherited Metabolic Disorders

• Hurler Syndrome (Mucopolysaccharidosis Type 1)
• Metachromatic Leukodystrophy (MLD)
• X-Linked Adrenoleukodystrophy (X-ALD)
• Other Inborn Errors of Metabolism