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Table 3 Reports of allogeneic stem cell transplantation (allo-SCT) for XLA

From: Allogeneic stem cell transplantation for X-linked agammaglobulinemia using reduced intensity conditioning as a model of the reconstitution of humoral immunity

 

Case number

Preconditioning

GVHD prophylaxis

Neutrophil engraftment

Increase of CD19+ and Ig

Reference

Howard et al.

N = 1

(−)

(−)

No

No

[6]

N = 3

(−)

CsA/MMF

No

No

[6]

Abu-Arja et al.

N = 1

ETP/CY/12Gy

TAC/MTX

Yes

Yes

[7]

Ikegame et al.

N = 1

FLU/CY/ATG/3Gy

CsA/MMF

Yes

Yes

This report

  1. There are two English reports of allo-SCT for XLA. Howard et al. present six cases based on their experience, three of which underwent SCT without preconditioning or GVHD prophylaxis, while the other three underwent GVHD prophylaxis consisting of cyclosporine and mycophenolate mofetil (CsA/MMF). No patients achieved donor engraftment or increases in CD19+ cell numbers or immunoglobulin (Ig) levels [6]. Abu-Arja et al. reported successful allo-SCT in a patient with XLA and AML, in which they used a myeloablative conditioning regimen consisting of etoposide 40 mg/kg for 1 day, cyclophosphamide (CY) 60 mg/kg/day, and 12 Gy of total body irradiation (ETP/CY/12Gy), and GVHD prophylaxis consisting of tacrolimus and methotrexate (TAC/MTX). The patient achieved engraftment with donor chimerism and had normal CD19+ cell numbers and Ig levels. In our case, the preconditioning regimen used comprised fludarabine, CY, rabbit anti-thymocyte globulin, and 3 Gy of TBI (FLU/CY/ATG/3Gy), and GVHD prophylaxis was CsA and MMF (CsA/MMF). As described in the text, the patient achieved engraftment with donor chimerism, with a rapid increase in CD19+ cell numbers and the production of Ig. To the best of our knowledge, this is the first English case report of successful allo-SCT for XLA