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I received an alert today about a new journal article that was released last week. "Hematopoietic Stem Cell Therapy for Multiple Sclerosis: Top 10 Lessons Learned" (Neurotherapeutics, 2012 Nov 29, Atkins and Freedman), an analysis of the HSCT research performed to date. Here are the highlights.
EFFECTIVENESS: "The use of higher intensity conditioning regimens, such as BEAM with ATG or busulphan/cyclophosphamide with ATG, is effective at suppressing episodic CNS inflammation. Among patients having relapses until the time of HSCT, more than 85 % who received a conditioning regimen of BEAM plus ATG were free from clinical relapses in the absence of ongoing treatment with other disease-modifying agents [50, 51]. Similarly, gadolinium-enhancing CNS lesions or new lesions on serial magnetic resonance imaging were uncommon [52, 53]." ... "The duration of stability in objective disability measures has been remarkable and appears to represent a marked change in outcome when compared to historical and concurrent MS populations."
SAFETY: "With time, there has been a marked reduction in transplant-related mortality. Indeed, the treatment-related mortality has decreased from 7.3 % for the period between 1995 and 2000 to 1.3 % during the period from 2000 to 2007 [20]. The reduction in mortality can be attributed to the selection of patients with better overall performance status for HSCT, a variable known to correlate with regimen-related mortality [43], combined with improvements in supportive care and the increasing experience of caring for MS patients at transplant centers."
COST: "The first study to address the economics of HSCT for MS plugged outcome data from case-matched SPMS patients who were treated with mitoxantrone or HSCT into a Markov-based economic model. The estimated cost of HSCT was less than £3000 (or $4700) per quality-adjusted life year [65]. Assumptions regarding treatment outcomes, such as the risk of mortality, the effectiveness in producing sustained stability in the EDSS and in procedural costs, influenced the outcome of the analysis. A variety of assumptions were made that tended to be biased against HSCT, including a relatively high regimen-related mortality of 5.3 % and the exclusion of costs associated with serious mitxoantrone-related complications, such as secondary acute leukemia. Notwithstanding these factors, HSCT was cost effective, using a yardstick of other treatments considered to be cost-effective in the United Kingdom. For comparison, current MS drug therapy cost effectiveness has been estimated to be between $80,000 to $168,000 per avoided relapse [66, 67] or upwards of $73,000 per incremental quality-adjusted life year [68, 69]."
CONCLUSION: "When the overall worldwide experience is viewed together, HSCT appears most beneficial for patients with highly active MS who are progressing and who are refractory to conventional MS therapies. HSCT can be performed with acceptable treatment-related morbidity and little mortality. Selected subpopulations, such as those patients with malignant MS, may benefit even more substantially from HSCT." ... "HSCT performed for patients with severe MS for the last 15 years has shown us the usefulness of this treatment. More importantly, it has provided a new treatment paradigm in which the immune system is repaired rather than suppressed."
Full Article:
http://link.springer.com/article/10....ext.html#Sec10
EFFECTIVENESS: "The use of higher intensity conditioning regimens, such as BEAM with ATG or busulphan/cyclophosphamide with ATG, is effective at suppressing episodic CNS inflammation. Among patients having relapses until the time of HSCT, more than 85 % who received a conditioning regimen of BEAM plus ATG were free from clinical relapses in the absence of ongoing treatment with other disease-modifying agents [50, 51]. Similarly, gadolinium-enhancing CNS lesions or new lesions on serial magnetic resonance imaging were uncommon [52, 53]." ... "The duration of stability in objective disability measures has been remarkable and appears to represent a marked change in outcome when compared to historical and concurrent MS populations."
SAFETY: "With time, there has been a marked reduction in transplant-related mortality. Indeed, the treatment-related mortality has decreased from 7.3 % for the period between 1995 and 2000 to 1.3 % during the period from 2000 to 2007 [20]. The reduction in mortality can be attributed to the selection of patients with better overall performance status for HSCT, a variable known to correlate with regimen-related mortality [43], combined with improvements in supportive care and the increasing experience of caring for MS patients at transplant centers."
COST: "The first study to address the economics of HSCT for MS plugged outcome data from case-matched SPMS patients who were treated with mitoxantrone or HSCT into a Markov-based economic model. The estimated cost of HSCT was less than £3000 (or $4700) per quality-adjusted life year [65]. Assumptions regarding treatment outcomes, such as the risk of mortality, the effectiveness in producing sustained stability in the EDSS and in procedural costs, influenced the outcome of the analysis. A variety of assumptions were made that tended to be biased against HSCT, including a relatively high regimen-related mortality of 5.3 % and the exclusion of costs associated with serious mitxoantrone-related complications, such as secondary acute leukemia. Notwithstanding these factors, HSCT was cost effective, using a yardstick of other treatments considered to be cost-effective in the United Kingdom. For comparison, current MS drug therapy cost effectiveness has been estimated to be between $80,000 to $168,000 per avoided relapse [66, 67] or upwards of $73,000 per incremental quality-adjusted life year [68, 69]."
CONCLUSION: "When the overall worldwide experience is viewed together, HSCT appears most beneficial for patients with highly active MS who are progressing and who are refractory to conventional MS therapies. HSCT can be performed with acceptable treatment-related morbidity and little mortality. Selected subpopulations, such as those patients with malignant MS, may benefit even more substantially from HSCT." ... "HSCT performed for patients with severe MS for the last 15 years has shown us the usefulness of this treatment. More importantly, it has provided a new treatment paradigm in which the immune system is repaired rather than suppressed."
Full Article:
http://link.springer.com/article/10....ext.html#Sec10
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