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Response to Brazzola et al

Bone Marrow Transplantation volume 36, page 1105 (2005)Cite this article

We read with great interest the report of Brazzola et al describing the unfortunate case of a patient with dyskeratosis congenita who died from intracranial bleeding 48 days posthematopoietic stem-cell transplantation from a matched unrelated donor. At that time the patient was still thrombocytopenic and requiring platelet transfusions. At autopsy the patient was diagnosed with diffuse capillaritis involving multiple organs including the brain.

Apparently, the reduced-intensity regimen incorporating fludarabine, cyclophosphamide and antithymocyte globulin did not prevent this complication, which has been observed in patients with dyskeratosis congenita after hematopoietic stem-cell transplantation. This observation is in contrast to the encouraging results using reduced-intensity hematopoietic stem-cell transplantation published by others1, 2 and us.3 As the authors conclude, it is indeed possible that the toxicity of such preparatory regimens is still not sufficiently low to decrease the morbidity in certain patients with dyskeratosis congenita undergoing transplant. However, it would be interesting to know whether besides the use of methotrexate, there were additional differences between the previously published successful cases and the patient in the present report. As this patient had multiple transfusions before the transplant and developed myelodysplasia, did he have pretransplant organ disease and iron overload? Also, did he have any vascular skin lesions to suggest that capillaritis was not only a transplant-related complication, but a part of the spectrum of dyskeratosis congenita? Last, how severe was the thrombocytopenia at the time of death, and could the bleeding be related to thrombocytopenia rather that to the vasculopathy? It is difficult to draw definite conclusions from small series of patients, but in rare disorders publications such as that of Brazolla et al are vitally important and may suggest specific risk factors for hematopoietic stem-cell transplantation, which help further refinement of the transplant regimen or indicate earlier transplantation before progression to more advanced disease.

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References

  1. Gungor T, Corbacioglu S, Storb R, Seger RA . Nonmyeloablative allogeneic hematopoietic stem cell transplantation for treatment of Dyskeratosis congenita. Bone Marrow Transplant 2003; 31: 407–410.

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  2. Nobili B, Rossi G, De Stefano P et al. Successful umbilical cord blood transplantation in a child with dyskeratosis congenita after a fludarabine-based reduced-intensity conditioning regimen. Br J Haematol 2002; 119: 573–574.

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  1. Division of Hematology/Oncology and Bone Marrow Transplantation, Marrow Failure and Myelodysplasia Program, The Hospital for Sick Children, The University of Toronto 555 University Avenue, Toronto, M5G 1X8, Ontario, Canada

    Y Dror

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Dror, Y. Response to Brazzola et al. Bone Marrow Transplant 36, 1105 (2005). https://doi.org/10.1038/sj.bmt.1705174

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