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Allogeneic bone marrow transplantation restores IGF-I production and linear growth in a γ-SCID patient with abnormal growth hormone receptor signaling

Bone Marrow Transplantation volume 33, pages 773–775 (2004)Cite this article

Summary:

Severe combined immunodeficiency (SCID) is a heterogeneous group of disorders characterized by a severe defect of both T- and B-cell immunity, which generally require allogeneic bone marrow transplantation (BMT) within the first years of life. We previously reported a patient affected with an X-linked SCID due to L183S hemizygous missense γ chain mutation, whose severe short stature was due to a peripheral growth hormone (GH) hyporesponsiveness associated to abnormal GH receptor (GH-R) signal transduction. In this study, we report the effect of BMT on the GH-R/insulin-like growth factor I (IGF-I) axis. After BMT, the patient showed a significant improvement in linear growth and normalization of basal- and GH-stimulated IGF-I values, which paralleled a fully competent immunological reconstitution. This suggests that cells derived from the hematopoietic stem cell may exert an unexpectedly significant role in producing IGF-I. This may also suggest that stem cell-based therapies may be useful for the correction of non-hematopoietic inherited disorders, such as those of GH-R/IGF-I axis.

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Acknowledgements

This study was supported by a grant from MIUR Prin 2002, Rome, Italy, and from Regione Campania, legge 502.

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Authors and Affiliations

  1. Department of Pediatrics, ‘Federico II’ University, Naples, Italy

    M Salerno, R Busiello, V Esposito, M Adriani & C Pignata

  2. Immunohematology Unit, ‘Federico II’ University, Naples, Italy

    E Cosentini

  3. Division of Hematology, ‘Federico II’ University, Naples, Italy

    C Selleri & B Rotoli

Authors
  1. M Salerno
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  2. R Busiello
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  3. V Esposito
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  4. E Cosentini
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  5. M Adriani
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  6. C Selleri
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  7. B Rotoli
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  8. C Pignata
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Correspondence to C Pignata.

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Salerno, M., Busiello, R., Esposito, V. et al. Allogeneic bone marrow transplantation restores IGF-I production and linear growth in a γ-SCID patient with abnormal growth hormone receptor signaling. Bone Marrow Transplant 33, 773–775 (2004). https://doi.org/10.1038/sj.bmt.1704421

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  • DOI: https://doi.org/10.1038/sj.bmt.1704421

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