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Successful allo-SCT in a Fanconi anemia patient with renal impairment using reduced doses of CY and fludarabine

Bone Marrow Transplantation volume 45, pages 415–416 (2010)Cite this article

BM failure is the main cause of morbidity in patients with Fanconi anemia (FA), but the presence of other abnormalities such as renal/urogenital anomalies (estimated to be present in about 24–30% of FA patients) can further aggravate the clinical course and affect the outcome. Patients may have complete absence of one kidney, hypoplastic, dysplastic, malformed (usually horseshoe) or ectopic kidneys, and double ureters. Any of the above problems can affect the renal function and may potentially lead to a state of renal failure.1

The literature available regarding SCT in FA patients with renal impairment is scarce. Heitink-Polle et al.2 recently reported a successful allo-SCT in a patient with FA and renal failure using a conditioning regimen of fludarabine and thoraco-abdominal irradiation, and we would like to describe here an alternative conditioning regimen without the use of radiation in a 2-year-old girl with confirmed FA who underwent matched related SCT at our institution. Before SCT, the patient was blood- and plt transfusion-dependent. In addition, she had evidence of chronic renal failure and was followed by the nephrology team; her renal failure was because of renal dysplasia secondary to left vesicoureteral reflux. On account of her increased requirements of plt and blood transfusions, a decision was made to proceed with SCT as at that point her renal failure was not deemed severe enough to warrant a renal transplant. A pre-SCT BM biopsy confirmed aplasia and showed no evidence of myelodysplasia, and no chromosomal abnormalities were noted on the cytogenetic analysis.

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Authors and Affiliations

  1. Department of Pediatric Hematology-Oncology, Section of Pediatric Stem Cell Transplant, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia

    W Turkistani & M Ayas

  2. Department of Pharmacy, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia

    M Ashour

  3. Department of Pediatrics, Section of Nephrology, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia

    H Al-Mojalli

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  1. W Turkistani
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  2. M Ashour
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Correspondence to M Ayas.

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Turkistani, W., Ashour, M., Al-Mojalli, H. et al. Successful allo-SCT in a Fanconi anemia patient with renal impairment using reduced doses of CY and fludarabine. Bone Marrow Transplant 45, 415–416 (2010). https://doi.org/10.1038/bmt.2009.162

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