Current Odds of Use

The best source for estimating the odds of receiving a stem cell transplant is Nietfeld and colleagues, who in 2008 published what has come to be known as the “Lifetime Probabilities Paper.” In this study, the researchers based their projections on HSCT data compiled by the Center for International Blood and Marrow Transplant Research (CIBMTR) for patients aged ≤70 years (considered the maximum age for a transplant) who underwent HSCT for any indication in the United States from 2001 to 2003.

Lifetime Probabilities Paper

Data on the incidence of malignancies commonly treated with HSCT were obtained from the US Surveillance, Epidemiology, and End Results (SEER) Program of the National Cancer Institute, which mines databases that encompass approximately 26% of the US population. SEER data do not include nonmalignant HSCT indications. Nietfeld et al used the SEER 13 database, which is built around patients diagnosed with cancer from 1992 to 2002. The analysis only included the diagnoses accounting for > 5% of the HSCTs in the CIBMTR database—that is, diseases for which transplants are rarely performed were not considered. Separate analyses of incidence were performed based on patient age.53

Four Possible Scenarios to Assess Probability of HSCT

Four possible scenarios were developed for this analysis to assess the probability of an HSCT that is: 

  1. autologous under current indications; 
  2. allogeneic with universal donor availability under current indications; 
  3. either autologous or allogeneic with universal donor availability; and  
  4. either autologous or allogeneic with universal donor availability and the expansion of current indications so that 50% of the patients with cancers that are treatable with HSCT receive a transplant.53

Assumptions Made in Calculating Probablilites

Several assumptions were made in calculating the probabilities: 

  1. only diagnoses currently indicated for HSCT were included; 
  2. universal donor availability was assumed with no restriction in the availability of an HLA-identical sibling (or comparable) donor; and 
  3. a transplant utilization rate of 15% to 20% was assumed based on the current rate of transplant associated with the specific diagnoses.54

Findings...

Basing their calculations on current conditions and indications, the researchers projected a probability of 1 in 1666 for autologous and allogeneic transplants by age 20 and a probability of 1 in 217 by age 70.53,54 When you compare these odds with those for being diagnosed with common disease such as Down syndrome or spina bifida, you see the odds of receiving a stem cell transplant are not all that long.