‣ Patient:

⁃ Ages 0-22 years at time of enrollment

⁃ Diseases:

∙ Patients with severe or very severe acquired AA, defined by:

⁃ Bone marrow biopsy demonstrating cellularity of \<25% (at least 2 weeks from last dose of G-CSF), in addition to 2 of the following: absolute neutrophil count (ANC) \<500/µL, platelets \< 20,000/µL and absolute reticulocytes \<40,000/µL

• Negative evaluation for inherited bone marrow failure conditions and negative evaluation for dysplasia or cytogenetic abnormalities associated with myelodysplastic syndromes

• Patients with concurrent paroxysmal nocturnal hemoglobinuria (PNH) clones are eligible, as long as they meet criteria for severe or very severe aplastic anemia as defined above

‣ Patients with clinically diagnosed and/or genetically proven iBMF syndromes, resulting in chronic red blood cell or platelet-transfusion dependence and/or an absolute neutrophil count \<500/µL. These disorders include, but are not limited to:

⁃ Fanconi Anemia

• Dyskeratosis Congenita

• Severe Congenital Neutropenia

• Diamond-Blackfan Anemia

• Congenital Dyserythropoietic/Sideroblastic Anemias

• Congenital Amegakaryocytic Thrombocytopenia

• Shwachman-Diamond Syndrome

⁃ Lansky or Karnofsky performance \>60

⁃ HLA matched related donor available.

⁃ No active untreated infection

⁃ Females of childbearing potential must have negative pregnancy test.

‣ Organ Function:

• Serum creatinine \<1.5xupper limit of normal for age Hepatic: Transaminases \<5x normal

• Cardiac shortening fraction \>27%

• Bilirubin \<2.5x normal (unless elevation due to Gilberts disease).

‣ Donor Selection Criteria:

• Donor selection will comply with U.S. Food and Drug Administration's Code of Federal Regulations

• Fully HLA-matched related donor.

• Donor must be at least 6 months of age

• Donor suitable for bone marrow collection and meets eligibility for donation, including fulfilling infectious disease criteria as per SOP, including HIV, Hepatitis B, Hepatitis C Polymerase chain reaction (PCR) negative.

• If subject has confirmed iBMF syndrome, donor must be evaluated for this disorder and testing must be negative

• Children's Hospital of Philadelphia (CHOP) bone marrow transplant (BMT) procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases.

• Donor evaluation and collection procedure as per CHOP Standard Operating Procedures (SOP)