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From ECTRIMS 2011: The dichotomous model for evaluation of treatment outcomes in multiple sclerosis patients after autologous haematopoietic stem cell transplantation
A.A. Novik, T.I. Ionova, G.V. Gorodokin, S.S. Salek for the European PRO Task Force
Efficacy of MS treatment is traditionally based on objective clinical data. At present patient-reported outcomes (PRO) are of increasing importance in evaluation of treatment outcomes in clinical medicine. Recently, a dichotomous model for evaluation of treatment outcomes in patients with a variety of internal diseases was proposed by the experts of the European PRO Task Force. This dichotomous model includes two arms: objective clinical data and PRO (quality of life, symptoms and others). We aimed to test this model in the MS population undergoing high-dose immunosuppressive therapy (HDIT) with AHSCT.
73 MS patients (secondary progressive – 23, primary progressive –14, progressive-relapsing – 1 and relapsing-remitting – 35) were included in this study (mean age - 32.5, range: 17-51; male/female – 32/41). Median EDSS at base-line was 3.5 (range 1.5 – 8.0). All the patients underwent HDIT+AHSCT. QoL was assessed using the generic questionnaire SF-36. QoL assessment and neurological examination were performed at baseline, at discharge, at 3, 6, 9, 12 months, and every 6 months thereafter. QoL treatment response was classified as improvement, stabilization or worsening. The mean follow-up duration was 24 months (range 18 – 48 months).
At 6 months after HDIT+AHSCT significant improvement of all the domains of SF-36 except pain and role emotional functioning as compared with base-line was demonstrated (p<0.05). At longer term follow-up further QoL improvement was found. At 6 months post-transplant QoL improvement was achieved in 52% of patients, QoL stabilization – in 36%, and QoL worsening - in 12% of patients. At long-term follow-up QoL improvement was registered in 44%, QoL stabilization – in 40% of patients, and QoL worsening – in 16% of patients. Clinical response in terms of EDSS changes at 6 months post-transplant was achieved in all the patients: 49% of patients had clinical improvement, 51% were stable. At long-term follow-up 54% of patients improved, 39% -stabilized, and 7% - worsened.
The findings of this study indicate that systematic evaluation of patients' clinical and QoL data allows us to obtain comprehensive information about treatment outcomes in MS patients undergoing HDIT+AHSCT. The dichotomous model appears feasible to measure the effect of treatment both from physician’s and patient’s perspective. The evaluation of both clinical and QoL response provides comprehensive information about the benefits and risks of treatment for MS patients.
A.A. Novik, T.I. Ionova, G.V. Gorodokin, S.S. Salek for the European PRO Task Force
Efficacy of MS treatment is traditionally based on objective clinical data. At present patient-reported outcomes (PRO) are of increasing importance in evaluation of treatment outcomes in clinical medicine. Recently, a dichotomous model for evaluation of treatment outcomes in patients with a variety of internal diseases was proposed by the experts of the European PRO Task Force. This dichotomous model includes two arms: objective clinical data and PRO (quality of life, symptoms and others). We aimed to test this model in the MS population undergoing high-dose immunosuppressive therapy (HDIT) with AHSCT.
73 MS patients (secondary progressive – 23, primary progressive –14, progressive-relapsing – 1 and relapsing-remitting – 35) were included in this study (mean age - 32.5, range: 17-51; male/female – 32/41). Median EDSS at base-line was 3.5 (range 1.5 – 8.0). All the patients underwent HDIT+AHSCT. QoL was assessed using the generic questionnaire SF-36. QoL assessment and neurological examination were performed at baseline, at discharge, at 3, 6, 9, 12 months, and every 6 months thereafter. QoL treatment response was classified as improvement, stabilization or worsening. The mean follow-up duration was 24 months (range 18 – 48 months).
At 6 months after HDIT+AHSCT significant improvement of all the domains of SF-36 except pain and role emotional functioning as compared with base-line was demonstrated (p<0.05). At longer term follow-up further QoL improvement was found. At 6 months post-transplant QoL improvement was achieved in 52% of patients, QoL stabilization – in 36%, and QoL worsening - in 12% of patients. At long-term follow-up QoL improvement was registered in 44%, QoL stabilization – in 40% of patients, and QoL worsening – in 16% of patients. Clinical response in terms of EDSS changes at 6 months post-transplant was achieved in all the patients: 49% of patients had clinical improvement, 51% were stable. At long-term follow-up 54% of patients improved, 39% -stabilized, and 7% - worsened.
The findings of this study indicate that systematic evaluation of patients' clinical and QoL data allows us to obtain comprehensive information about treatment outcomes in MS patients undergoing HDIT+AHSCT. The dichotomous model appears feasible to measure the effect of treatment both from physician’s and patient’s perspective. The evaluation of both clinical and QoL response provides comprehensive information about the benefits and risks of treatment for MS patients.
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