- The cost and
effectiveness of gene therapy was analyzed against hematopoietic stem cell
- The results
indicate that while gene therapy was more expensive, it was also a lot
less complicated and more effective and safe
Gene therapy is safer and effective than hematopoietic stem cell
transplantation (HSCT) for
curing beta-thalassemia, reveals a new study.
there a Cure for Beta-thalassemia?
Till date, there is no
genetic cure for a hereditary blood disorder, beta-thalassemia where the globin
gene is defective and does not produce adequate hemoglobin for the body's
needs. As a result, patients need to be on a lifelong regimen of monthly blood
transfusions and iron chelation therapies. Even with treatment, there are
several risks and co-morbidities associated with the disease such as the
transmission of viruses through blood transfusions and iron overload in vital
The only available
curative for this condition is allogeneic
hematopoietic stem cell transplantation (HSCT)
with a matched donor. This
is not an easy process or procedure and nearly 75 percent of all thalassemia
patients do not have an HLA (human leukocyte antigen) matched sibling.
‘Gene therapy is more safe and effective for curing beta-thalassemia. Though it is more expensive than hematopoietic stem cell transplantation (HSCT), gene therapy is less complicated.’
Finding a matched, the
unrelated donor is only a recent development with the establishment of bone
marrow registries and finding such a match is not really easy. Besides, age
factor also matters in the success of the transplanted marrow and despite all
care and caution; GVHD (graft versus host disease) is a major cause of
morbidity and deaths. Though scientists have been working on gene therapy for
decades, it is only since 2010 that new progressive strides were made in
successfully curing beta-thalassemia
involves the delivery of a lab-made normal globin gene via a delivery vehicle
which is a viral vector. This is infused into the patient's own stem cells
(collected before the procedure) and infused back into the patient with the
normal gene and delivery vector. This ensures the normal production of
hemoglobin adequate for the body functions. Gene therapy does not involve a
transplant since the patient's own stem cells are harvested and infused back
thus eliminating the risk of rejections. Besides, gene therapy can be safely
used on adults with minimal risks.
Curative Treatment of Beta Thalassemia
French researchers have
published a paper titled "Innovative Curative Treatment of Beta Thalassemia:
Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell
Transplantation" analyzing the safety and efficacy of gene therapy over hematopoietic stem
(HSCT) as a curative.
The team did a
comparative study analyzing the cost and effectiveness of gene therapy as
against hematopoietic stem cell transplantation (HSCT). They looked at the data
and outcomes of seven patients treated between 2009 and 2016 of which four
received gene therapy and three received an HSCT.
The team compared
two-year costs and outcomes of patients treated with gene therapy and HSCT.
They looked at critical factors such as length of hospital stay, interim
hospitalizations, grade III and grade IV complications.
Patients treated with gene therapy were
older yet did not present with as many complications or hospitalizations.
Infections for patients treated with HSCT were three times higher than for gene
therapy. While the outcomes were more positive for gene therapy, costs were
The team estimated the
total costs from hospital accounts and national cost studies. They found that
gene therapy costs around €608,086 while HSCT costs around €215,571. The high
cost of gene therapy was mainly due to the procurement of the vector which
accounted for 48% of the total costs.
The end results indicate
that while gene therapy was more
expensive, it was also a lot less complicated and more effective and safe.
The study results are
published in the journal Human Gene
- Coquerelle, Severine, Mariem Ghardallou, Setti Rais, Pierre Taupin, Fabien Touzot, Laure Boquet, Stéphane Blanche et al. "Innovative curative treatment of βeta-thalassemia: cost-efficacy analysis of gene therapy versus allogenic hematopoietic stem cell transplantation." Human gene therapy (2019). Accessed on 21st April 2019 from - (https://doi.org/10.1089/hum.2018.178)