Table 1 Conditioning regimens.

From: EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

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A

   

Busulfan i.v (AUC = 85–95 mg*h/L) Fludarabine (160 mg/m2)

B

   

Treosulfan (30–42 g/m2) Fludarabine (150–160 mg/m2) Thiotepa (8–10 mg/kg)

C

  

Busulfan i.v. (AUC = 60–70 mg*h/L) Fludarabine (160–180 mg/m2)

 

D

  

Treosulfan (30–42 g/m2) Fludarabine (150–160 mg/m2)

 

E

 

Fludarabine (150–160 mg/m2) Melphalan (140 mg/m2)

  

F

Fludarabine (150 mg/m2) Cyclophosphamide (20–40 mg/kg)

   
  1. Protocol A and B: These are recommended for patients without severe preexisting organ damage and non-SCID diseases where a complete donor chimerism is desired for optimal disease correction.
  2. Protocols C and D: These are recommended for patients with preexisting organ damage and/or diseases where engraftment has been shown to reliably occur with reduced intensity conditioning. Mixed donor chimerism is more likely to occur compared to protocols A and B.
  3. Protocol E: This may be best suited for patients with preexisting organ damage and/or diseases where full myeloid engraftment is not absolutely required. Higher degrees of chimerism can be achieved when using PBSC. DLI may be required in case of mixed chimerism.
  4. Protocol F: To avoid organ toxicity this regimen is only recommended for patients with DNA repair/radio-sensitivity disorders (except Artemis deficiency) in which alkylating agents are used in low dose.
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