Allogeneic stem cell transplantation in Fanconi anemia patients presenting with myelodysplasia and/or clonal abnormality: update on the Saudi experience

Ayas, Mouhab; Al‐Jefri, Abdullah; Al-Seraihi, Amal; Al-Mahr, Mohammed; Rifai, Samira; Al-Ahmari, Ali; Khairy, Ashraf; Elhassan, Ibrahim M.; El-Solh, H

doi:10.1038/sj.bmt.1705903

Cited by 16 publications

(13 citation statements)

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“…In 2008, Ayas et al reported 11 patients with FA with MDS and/or clonal abnormalities, including 1 patient with clear AML (4 patients had only a cytogenetic clone), who were transplanted using cyclophosphamide and total body irradiation (TBI; 450 cGy). 46 Ten patients were reported alive with no evidence of disease with a median FU of nearly 4 years. The overall excellent results illustrate the possibility of long-term survivors after HSCT in this situation.…”

Section: Outcome Current Management and Treatmentmentioning

confidence: 99%

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How I treat MDS and AML in Fanconi anemia

Latour

Soulier

2016

Blood

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Fanconi anemia (FA) is the most frequent inherited cause of bone marrow failure (BMF). Most FA patients experience hematopoietic stem cell attrition and cytopenia during childhood, which along with intrinsic chromosomal instability, favor clonal evolution and the frequent emergence in their teens or young adulthood of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). To early identify and further predict bone marrow (BM) clonal progression and enable timely treatment, the follow-up of FA patients includes regular BM morphological and cytogenetic examinations. Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment of FA patients with MDS or AML. Although questions remain concerning HSCT itself (including the need for pretransplant chemotherapy, the best conditioning regimen, and the optimal long-term follow-up of such patients especially regarding secondary malignancies), clonal evolution in the absence of significant BM dysplasia and blast cells can be difficult to address in FA patients, for whom the concept of preemptive HSCT is discussed. Illustrated by 3 representative clinical vignettes showing specific features of MDS and AML in FA patients, this paper summarizes our practical approach from diagnosis through treatment in this particular situation.

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Section: Outcome Current Management and Treatmentmentioning

confidence: 99%

“…Outcomes in FA patients with MDS or AML Three single-center studies of more than 10 patients with FA and MDS/ AML 16,33,46 and 2 large multicenter studies 14,15 have been published to date with a reasonable FU.…”

Section: Outcome Current Management and Treatmentmentioning

confidence: 99%

How I treat MDS and AML in Fanconi anemia

Latour

Soulier

2016

Blood

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“…18 In the same way, the experience reported in Saudi Arabia appeared interesting after conditioning containing CY, antithymocyte globulin and TBI at 450 cGy for FA patients with myelodysplasia. 19 Finally, drugs associated with fludarabine and addition of ionizing irradiations still remain to be discussed, according to donor type (either related or unrelated) and/or according to marrow clonal abnormality occurrence before transplantation. In 2006, Bitan et al 20 reported excellent results regarding seven patients who underwent HSCT from either matched sibling or matched unrelated donor after fludarabine-based reduced-intensity conditioning regimen without radiation.…”

Section: Current Resultsmentioning

confidence: 99%

HSCT for Fanconi anemia in children: factors that influence early and late results

Dalle

2008

Bone Marrow Transplant

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Fanconi anemia (FA) is a rare autosomal recessive disease characterized by congenital abnormalities, cancer predisposition and progressive BM failure. FA patients present spontaneous and induced chromosome breakage. Hematopoietic SCT (HSCT) represents the unique therapeutic option to restore normal hematopoiesis when marrow failure or clonal hematopoietic abnormality occurs. Conventional myeloablative conditioning regimen, especially including a high dose of irradiation, appeared strongly toxic for FA patients. Then, reduced-intensity conditioning regimens were developed successfully for those patients. However, TRM still remained higher than for other HSCT indications. The development of fludarabine containing a non-myeloablative conditioning regimen appears to be a major progress. Long-term follow-up is absolutely necessary.

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“…6 Thus, we and others have advocated more intense conditioning regimens using TBI for FA patients presenting with such findings. 1,7 Despite the fact that second transplants may be successful in those FA patients who fail their first SCT, 8 the graft failure associated with recurrence of MDS/ leukemia or clonal chromosomal changes may be more difficult to manage and consequently the outcome may be less satisfactory and only few such cases have been reported in the literature. Tan et al 9 described a patient with MDS who relapsed almost 5 months after the first SCT (conditioning was with CY/fludarabine/ATG).…”

mentioning

confidence: 97%