On the 14th of August of 2018, the Norwegian Institute of Public Health (NIPH) has published a health technology assessment report in relation to autologous hematopoietic stem cell transplantation (AHSCT) comparing with standard treatment for patients with systemic sclerosis. Initially, the report was commissioned to NIPH by Ordering Forum (Bestillerforum RHF) as AHSCT has been suggested for a small group of patients with diffuse systemic sclerosis who have insufficient effect of standard pharmacological treatment to prevent serious and irreversible organ damage. The objective of the report is to perform a systematic review of efficacy and safety, an economic evaluation and ethical evaluation related to AHSCT for systemic sclerosis.
The key messages identified by the NIPH are provided below:
- Transplant‐related mortality was between seven and ten percent after AHSCT (low confidence in the effect estimate);
- Total mortality and organ failure rates at two years and beyond were lower in the AHSCT group than in the standard treatment group (moderate confidence in the result);
- AHSCT resulted in more side effects and adverse events including viral infections than standard treatment measured after two and six years (moderate confidence in the effect estimates);
- Skin involvement improved after AHSCT measured two and five years after treatment (moderate confidence in the result);
- Lung function measured by forced vital capacity and total lung capacity also improved to a certain extent after AHSCT, while the diffusion capacity for carbon monoxide (DLCO) and residual volume were not different from standard treatment measured two and five years after treatment (moderate confidence in the effect estimate for DLCO, and low for the others);
- AHSCT improved self‐perceived physical health measured two and five years after treatment (moderate confidence in the effect estimates);
- Costs associated with AHSCT in the treatment‐year are approximately 600,000 kroner per year per patient;
- AHSCT is ethically challenging as it may lead to premature death, but it may also lead to improved function and may be life‐prolonging.
The NIPH came to the following conclusions:
- The transplant‐related mortality after AHSCT is high, but risk of organ failure and death, for whatever reason, is nevertheless lower after two years follow‐up and beyond compared to standard treatment;
- AHSCT provides a significant improvement of skin involvement and lung function measured by forced vital capacity compared to standard treatment with cyclophosphamide injections two to five years after initiation of treatment, but side effects and adverse events occur more frequently than in standard treatment;
- The confidence in the quality of the evidence is low and moderate, mainly due to the limited number of patients in the studies, and that blinding is not possible in this type of study.
See the main results of the assessments in Norwegian and English here.
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