Grandmother’s stem cells cure rare bleeding disorder

Doctors at a private super speciality hospital here have claimed to have successfully cured a rare bleeding disorder in a four-year-old boy using stem cells from his grandmother.

Tarun, who comes from a family where consanguineous marriages are common, had been diagnosed with glanzmann's
thrombasthenia at the age of ten months when he was evaluated
for severe bleeding from nose and vomitting of blood, requiring blood transfusion, a release issued by Narayana Hrudayala said here.

This disease is a rare bleeding disorder which is genetically acquired and is autosomal recessive in nature. Less than 100 cases have been reported from India so far with the largest series of 42 cases from South India.

Glanzmann’s thrombasthenia is a genetic defect in the platelets, due to the absence of a receptor, as a result of which, blood does not clot, even though the platelet numbers are normal.

Tarun had  been having recurrent episodes of severe bleeding from the nose with vomiting of blood leading to severe anaemia, requiring blood transfusions. 

A bone marrow treatment using stem cells of his father failed, the release said. 

A doctors searched other family members for a matched donor and found that Tarun's grandmother's stem cells fully matched.

The child, hence, underwent a second transplant using the grandmother’s stem cells and engrafted successfully. Post
transplant, blood tests have confirmed that 100% of the blood cells are the grandmother’s and the platelet function tests have confirmed that the platelets are functional, the release said.