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Genetic and Rare Diseases Information Center (GARD)

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Fanconi anemia


Other Names for this Disease
  • Fanconi pancytopenia
  • Fanconi's anemia
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Your Question

I had Fanconi anemia and underwent chemothearpy and a bone marrow transplant as a young child. I am now a healthy young adult. Is there any data regarding life expectancy following a bone marrow transplant? I am aware of the increased risk for certain cancers. Also can Fanconi anemia and its treatment affect growth? If so, what treatment is available. Lastly, do you know why my doctor adivised me not to weight lift? 

Our Answer

We have identified the following information that we hope you find helpful. If you still have questions, please contact us.

Is there any data regarding life expectancy of children who underwent a bone marrow transplant? 

Allogeneic hematopoietic stem cell transplantation (HCT), a type of bone marrow transplant, has long been the primary treatment method for correcting the blood defects associated with Fanconi anemia.[1] In general, it has been estimated that five-year survivors of HCT may have a normal to near normal life expectancy,[2] however Fanconi anemia is a risk factor that negatively impacts survival rates. One study estimated a 58 percent 30-years survival rate for one-year survivors of HCT with Fanconi anemia.[1]
Last updated: 3/11/2013

Does Fanconi anemia and/or allogenic hematopoietic cell transplantation affect growth?

Yes. It is not uncommon for people with Fanconi anemia to have short stature. Also, allogenic hematopoietic cell transplantation (HCT) and total body irradiation can affect final height in transplanted children, particularly when the transplant is preformed prior to the age of five. Allogeneic HCT may also cause a decrease in lean body mass and a decline in body mass index after transplant.[3][4]
Last updated: 3/11/2013

What other long-term health complications are childhood survivors of allogeneic hematopoietic stem cell transplantation at risk for?

There are a number of long-term health risks associated with hematopoietic stem cell transplantation (HCT). Specific risks vary depending on a variety of factors, including age, gender, type of pre-transplant therapy, donor type, reason for HCT, and if the person experienced early complications (e.g., graft-versus-host disease or infections).[5] Examples of HCT related health risks include, blood cancer, solid tumors, heart disease, infection, lung toxicity, and chroinc graft-versus-host-disease.[5]  

HCT related risks appear to be higher in people with Fanconi anemia. Fanconi anemia makes the body especially sensitive to radiation and chemotherapy. The Fanconi Anemia Rearch Fund, Inc offer the Fanconi Anemia: A Handbook for Families and their Physicians (Third Edition, 2000) which provides detailed information on Fanconi anemia, including bone marrow transplantation. 
Last updated: 3/11/2013

Why should people with Fanconi anemia and/or a bone marrow transplant avoid weight lifting?

We recommend that you disucss this question with your healthcare provider directly.
Last updated: 3/11/2013

References
  • Sanders JE et al.,. Late effects among pediatric patients followed for nearly 4 decades after transplantation for severe aplastic anemia. Blood. 2011;118(5):1421; http://www.ncbi.nlm.nih.gov/pubmed/?term=21653322. Accessed 3/11/2013.
  • Cutler C. The approach to hematopoietic cell transplantation survivorship. In: Basow. UpToDate. Waltham, MA: UpToDate; 2012;
  • Freycon F et al. Final height and body mass index after fractionated total body irradiation and allogeneic stem cell transplantation in childhood leukemia. Pediatr Hematol Oncol. 2012;29(4):313; http://www.ncbi.nlm.nih.gov/pubmed/?term=22568794. Accessed 3/11/2013.
  • Inaba H et al. Longitudinal changes in body mass and composition in survivors of childhood hematologic malignancies after allogeneic hematopoietic stem-cell transplantation. J Clin Oncol. 2012 Nov;30(32):3991-7. Epub 2012 Oct 1; http://www.ncbi.nlm.nih.gov/pubmed/?term=23032628. Accessed 3/11/2013.
  • Cutler C. The approach to hematopoietic cell transplantation survivorship. In: Basow. UpToDate. Waltham, MA: UpToDate; 2012;