Childhood MDS and MPN treatment
How is childhood MDS and MPN treated?
Prognosis and treatment for children diagnosed with MDS is dependent on the specific type of MDS, the severity of the disease, whether the MDS is a primary or secondary and if there is an available stem cell donor. Some children with refractory cytopenia need only to be monitored with blood tests and may occasionally need blood transfusions to relieve symptoms, but their disease can remain stable for months or years.
Currently, an allogeneic (donor) stem cell transplant is the only long term curative option for MDS. The donor may be a matched sibling or an unrelated matched donor. This complex process replaces your child’s defective bone marrow, using high dose chemotherapy, then giving them a healthy donor’s stem cells.
A large proportion of children diagnosed with the MDS subtype of RC are cured after undergoing an allogeneic transplant. Fifty percent of children with RAEB and RAEB-T are also cured with a transplant.
For children with RC who are not eligible for an allogeneic transplant or are unable to find a matched donor, immunosuppressive therapy has shown some success, though further research is currently underway to examine this. This type of treatment can weaken a child’s general immune system and make them more susceptible to infections while undergoing the treatment.
Children diagnosed with a secondary type of MDS have less favourable outcomes compared to primary MDS, however allogeneic transplants have shown successful results in these children. Children with secondary MDS resulting from congenital diseases of the marrow may also be offered an allogeneic transplant as a treatment option.
Remember, however, that no two children are the same. In helping you to make the best treatment decision for your child, their doctor will consider all the information available including the details of your child’s particular situation.
Incidence of MPN in children is very rare and therefore treatment is based on a child-by-child basis, examining their diagnosis, genes affected and previous medical history.
Potentially effective treatments include chemotherapy and an allogeneic (donor) transplant. Treatment with other medications may also result in a temporary improvement of the disease. Occasionally, some children may have a more indolent disease and with supportive care such as blood transfusions, they may survive for many years without any active treatment.