Types of Blood and Immune Disorders

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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