Table 3 Haploidentical HSCT platforms.

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

 

TCR α/β [26, 27, 54]

PT-Cy [24, 25]

CD34 positive selection (only recommended for some SCID transplants)

Protocols

A, B, C, D

A, B, C, D

C, D

Graft

TCR α/β - CD19 depleted PBSC

unmanipulated bone marrow (1st choice) or PBSCa (2nd choice)

CD34 positive selected PBSC

Cell dose

10-20 x 10e6 CD34/kg

3–5 × 10e8 TNC/kg

10–20 × 10e6 CD34/kg

Serotherapy

ATG Grafalon: 3 × 4 mg/kg (d-4 to -2)b Rituximab: 200 mg/m2 (d-1)

Alemtuzumab: 2 × 0.2 mg/kg (d-10 to -9) If Alemtuzumab is not available: ATG Thymoglobuline 3 × 2.5 mg/kg (d-10 to -8)c

None

GVHD prophylaxis

If αβ T cells in graft ≥10e5/kg: add CSA

Cyclophosphamide 50 mg/kg on d + 3 and d + 4 Tacrolimus or CSA from d + 5 until at least d + 100 MMF from d + 5 to d + 35

none

  1. aIn case PBSC are used, higher rates of cGVHD can be expected and additional or prolonged GvHD prophylaxis may be considered [101].
  2. bIn case there is no access to ATG-Grafalon alternative serotherapy approaches (i.c. thymoglobulin) may be considered although published recommendations on optimal dose and timing are currently unavailable.
  3. cIn case of ATG in PT-Cy protocol additional Rituximab may be considered.
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