01 May 2019

Beta-thalassaemia patients have better outcomes after gene therapy than after stem cell transplant, according to a small single-centre study.

As most patients do not have a matching sibling to provide haematopoietic stem cells for transplantation, Dr Séverine Coquerelle of the Université Paris Diderot in France, and colleagues, looked at the cost and efficacy of gene therapy.

They compared outcomes and costs after two years for seven beta-thalassaemia patients, treated either with gene therapy (four patients) or haematopoietic stem cell transplantation (three patients) between 2009 and 2016, using hospital figures and national cost estimates.

The researchers report that at Necker Hospital in Paris, gene therapy was associated fewer complications, hospital admissions, and infectious complications.

The average cost for gene therapy was €608,086, versus €215,571 for stem cell transplant. Most of the extra cost for gene therapy was to provide the lentiviral vector needed to introduce the beta-globin gene into blood stem cells.

In the journal Human Gene Therapy, the authors write: “Gene therapy is a promising treatment that can be proposed to patients without human leukocyte antigen-matched siblings.”

They conclude that this treatment “was costlier but resulted in fewer complications than hematopoietic stem cell transplantation”.

Dr Terence Flotte, editor-in-chief of Human Gene Therapy, commented: “There has been much discussion and controversy about the high cost of gene therapy, but what has been lacking is a direct comparison to alternative therapies, which themselves are also often very costly and may produce suboptimal outcomes.

“This study does a critical comparison of both cost and outcomes for patients with beta-thalassaemia, whose primary alternative to gene therapy would be hematopoietic stem cell transplantation. These data may enable a more rational debate of the overall value of gene therapy for this relatively common genetic disease.”

Source: Coquerelle, S., Ghardallou, M., Rais, S., Taupin, P., Touzot, F., Boquet, L., Blanche, S., Benaouadi, S., Brice, T., Tuchmann-Durand, C., Ribeil, J.A., Magrin, E., Lissillour, E., Rochaix, L., Cavazzana, M., Durand-Zaleski, I. (2019) “Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation”, Human Gene Therapy, available from doi: 10.1089/hum.2018.178


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