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How truly risky is HSCT today?
The consent form I signed for HALT-MS listed the risk of death at 5%. But that form was written in 2006 when the study was created ... and based on earlier experiences. What is the true risk in 2012?
Earlier trials included two known-to-be-potentially fatal treatments no longer used in HSCT for MS: Total body irradiation (TBI) and the chemotherapy agent busulfan. Also, clinical trials so far have been small, so one death can make a big difference in the final study results. Even though there have been zero deaths in HALT-MS due to the treatment, if one member passes away we would be up to over 4%.
A long-term Greek study had two deaths (6%) that were the result of complications from the treatment, one at two months and another at 2.5 years. While BEAM was used in most cases, 10 patients received busulfan--including the two who died.
In a more recent Italian study with the last transplant in 2008, 2.7% died (again, two people) using a BEAM/ATG regimen. But the study started in 1996 and the procedure has gotten safer over the years as doctors are more aggressive at treating infections and hospitals are more aware of the necessity to keep everything sterile.
Another study (Novik et al) had one death (2%) that occurred after a sepsis infection on Day +8. Fassas in 2008 wrote that HSCT is "a rather toxic procedure associated with a mortality risk of 2-3%." Farge et al found about a 2% risk for people with MS. But Hugle et al reports "Transplant-related mortality following autologous HSCT has decreased significantly over the years due to growing experience and careful selection of patients."
The location where the HSCT is performed also make a difference. Farge et al: "In multivariate analysis, the 100-day transplant-related mortality ... was lower in experienced centers (P=0.003)."
Bottom line. Roland Martin sums it up best in his 2007 article "Is haematopoietic stem cell transplantation a treatment option for severe MS or not?" in the journal Brain. "Current transplant-related mortality is ∼1% and, in my view, acceptable in patients with severe and rapidly advancing disease."
http://brain.oxfordjournals.org/content/130/5/1181.full
I would agree with both the 1% and acceptable short- and long-term risk part. If done at an experienced center, the odds of death are relatively low. (Although it must be pointed out that mistakes at the best facilities do happen, and early life-threatening infections don't care what hospital you are at.)
Even without death there is still risk related to lifelong complications--some minor (like my low testosterone) and some major (like a patient losing toes and her hearing due to sepsis). These cases don't count against mortality but, especially in certain cases, are considerable factors when weighing the risk of this treatment. And long term risks from the chemotherapy, such as a higher risk of blood diseases years into the future, cannot be ignored.
With any aggressive treatment there is risk. How much is acceptable to you only you can decide.
Earlier trials included two known-to-be-potentially fatal treatments no longer used in HSCT for MS: Total body irradiation (TBI) and the chemotherapy agent busulfan. Also, clinical trials so far have been small, so one death can make a big difference in the final study results. Even though there have been zero deaths in HALT-MS due to the treatment, if one member passes away we would be up to over 4%.
A long-term Greek study had two deaths (6%) that were the result of complications from the treatment, one at two months and another at 2.5 years. While BEAM was used in most cases, 10 patients received busulfan--including the two who died.
In a more recent Italian study with the last transplant in 2008, 2.7% died (again, two people) using a BEAM/ATG regimen. But the study started in 1996 and the procedure has gotten safer over the years as doctors are more aggressive at treating infections and hospitals are more aware of the necessity to keep everything sterile.
Another study (Novik et al) had one death (2%) that occurred after a sepsis infection on Day +8. Fassas in 2008 wrote that HSCT is "a rather toxic procedure associated with a mortality risk of 2-3%." Farge et al found about a 2% risk for people with MS. But Hugle et al reports "Transplant-related mortality following autologous HSCT has decreased significantly over the years due to growing experience and careful selection of patients."
The location where the HSCT is performed also make a difference. Farge et al: "In multivariate analysis, the 100-day transplant-related mortality ... was lower in experienced centers (P=0.003)."
Bottom line. Roland Martin sums it up best in his 2007 article "Is haematopoietic stem cell transplantation a treatment option for severe MS or not?" in the journal Brain. "Current transplant-related mortality is ∼1% and, in my view, acceptable in patients with severe and rapidly advancing disease."
http://brain.oxfordjournals.org/content/130/5/1181.full
I would agree with both the 1% and acceptable short- and long-term risk part. If done at an experienced center, the odds of death are relatively low. (Although it must be pointed out that mistakes at the best facilities do happen, and early life-threatening infections don't care what hospital you are at.)
Even without death there is still risk related to lifelong complications--some minor (like my low testosterone) and some major (like a patient losing toes and her hearing due to sepsis). These cases don't count against mortality but, especially in certain cases, are considerable factors when weighing the risk of this treatment. And long term risks from the chemotherapy, such as a higher risk of blood diseases years into the future, cannot be ignored.
With any aggressive treatment there is risk. How much is acceptable to you only you can decide.
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