There are about 60,000 children in Pakistan suffering from thalassemia major, the most common fatal genetic disease in the world. Supportive care for this disease is a huge economic burden on affected families, being higher than the average income. As a consequence, the majority of children do not live beyond adolescence. The cost of bone marrow transplantation is equivalent to a few years of supportive therapy, but contrary to this, provides a definitive cure, returning to normal life over 80% of children who have a compatible sibling. This treatment modality is therefore necessary from a medical, ethical and economical standpoint.
The project began in 2008 thanks to he efforts of Dr Sadaf Khlaid and her husband Capt. (R) Zaman Khalid, parents of Ayesha, a young girl with thalassemia transplanted in Italy by Dr Faulkner’s team using maternal stem cells. Initial funding was provided by Fondazione Monte dei Paschi di Siena, Italy. The aim was to provide financial and professional support for at least six transplants for each Pakistan institution with the minimum requirements and the willingness to participate in the project: The National Institute for Blood Diseases (NIBD) in Karachi, the Pakistan Institute of Medical Sciences (PIMS) and SHIFA International Hospitals in Islamabad. Low-risk patients with thalassemia major from poor families were screened for the presence of a suitable donor and treated with a shared and common protocol that applies the best available therapeutic modality. The initial objective was to obtain cure with good quality of life in at least 80% of the children. This project includes a program to support the family throughout treatment (approx. 6 months) as well as screening and prevention for all of families involved.
In December 2010, C2C received a grant of 50 million PKR (approx. €450,000) for the Cure & Prevention of Thalassemia Major as part of the international debt-swap agreement between Italy and Pakistan (PIDSA – Pakistan Italian Debt-for development Swap Agreement).
As of March 2012 over 500 families have been registered and offered free screening, and a total of 70 children have undergone transplantation. In the 30 low risk cases the success rate was 87%, a result consistent with the original objective and comparable to that obtained in Italy and other rich countries. In higher-risk children success has been in the 62% range, which is also an acceptable result which is gradually improving.
These results have been obtained with a cost about ten times lower than that in rich countries, not considering all the avoided inconvenience and hardship for the families involved and the contribution to the growth and motivation of local doctors and nurses.
The support program for families has allowed safe and adequate living conditions and adherence to prescribed treatments after hospital discharge.