Você está aqui
Girl with metabolic disorder has life-saving cord blood transplant from her brother
Neha is now a healthy six year old following a cord blood transplant from her younger brother, made possible by a no-cost sibling program from the family cord blood bank Babycell (formerly named Babycell) in India.
By the age of one year old, Neha started missing major growth milestones that a normal baby would achieve in infancy, and that’s when her parents discovered that there is an abnormality. Neha was diagnosed with a rare metabolic disorder called mucopolysaccharidoses VI (MPS VI).
The MPS diseases are storage disorders where the patient’s body lacks lysosomal enzymes to properly break down carbohydrates in their cells. Babies with the MPS genetic defect seem normal at first, but as cellular damage accumulates they fall behind normal growth and develop a distinctive appearance. Common symptoms are short stature, skeletal dysplasia, motor dysfunction, heart defects, and visual difficulties. Patients with MPS metabolic disorders become progressively worse and do not survive past their second decade of life.
The diagnosis came as a big shock for the family and they were clueless as to how to help their little angel. Neha’s father Mahendra said "When we got the news our world stopped for a while. We met the doctor and they asked us to go for a stem cell transplant, but we were clueless if we would ever find a donor!"
It is only within the past decade that Dr. Kurtzberg’s group at Duke University demonstrated that storage disorders could be successfully treated with cord blood transplants. The cord blood transplant is able to correct the patients’ metabolism so that they create the enzymes they need to clean their cells and their neurological conditions improve.
After long discussions and endless doctor visits, Neha’s parents were asked to plan a second baby who could provide a cord blood transplant for their daughter. Little did they know about the treatment and concept of stem cells. Her father said, "We met representatives of several cord blood stem cell banks but they were only ready to offer banking service and not transplant assistance. When we met technical directors and cord blood transplant experts from Biocell, we saw a ray of hope. Biocell assisted us with collection, processing and preservation of our second baby’s cord blood stem cells, at no cost!"
Just a couple of years after her baby brother was born, the stem cells from his umbilical cord blood were transplanted into Neha in January 2016, at Apollo Hospital in Chennai, India. Neha has now passed her 200 days milestone post-transplant.
Neha has managed to talk fluently, and she is now physically and emotionally more responsive with her body showing steady growth. Her father said, "Neha is doing really well, she has coped with the transplant and has sailed through it all, it feels like a new birth of our daughter."
Regarding the banking experience Neha’s father said, "Thank you just does not cover it all, Babycell and the Doctor have given us unconditional support and we have no words to express our gratitude. Without them my daughter would not have survived for too long. There would have been a vacuum in our life forever if our daughter would not have been treated." Neha is enjoying her new life.