Doctors save the life of a child with a rare genetic blood disorder

ABU DHABI: Sheikh Khalifa Medical City (SKMC) has reported the story of a two-year-old boy with a rare genetic bleeding disorder Hemophilia A where the timely intervention by doctors helped avoided the need for a major neurosurgical operation.

Hemophilia A, an inherited bleeding disorder affecting mainly males, is characterised by low levels of factor VIII, a protein required for blood to clot. People with this disorder tend to bleed more than normal after an injury or undergoing a dental procedure or surgery, or mostly spontaneous bleeding mainly in large joints. This is particularly challenging for children living with the disorder, as it puts significant limitations on their daily lives, with the potential for a normal injury to become life-threatening. While there is currently no definitive cure, proper management and timely treatment can allow youngsters with Hemophilia A to enjoy life, like any other healthy child.

The two-year-old patient, called Saif, was brought to SKMC with extensive cerebellar and subdural hematoma (bleeding in the brain) and was known to have Hemophilia A. Following a new therapy and medical management by SKMC, Saif is now expected to lead a normal life.

Dr. Naser Alzein, Chief of Paediatric Hematology/Oncology, Sheikh Khalifa Medical City, said, “Our plan was to start Saif on a proper recombinant Factor VIII therapy in a different way to overcome difficulties to achieve our target of normal hemostasis to control the bleeding with no need of surgical intervention. This was followed by the new modality of therapy, Emicizumab, which is a humanised bispecific antibody for the treatment of Hemophilia A. We are extremely happy with Saif’s progress post-treatment, and that we could protect him from having to undergo major surgery. We are expecting him to lead a normal life and be able to perform regular activities, much like any other child his age. After being discharged from SKMC, he will be closely monitored by our team at the Paediatric Hematology Clinic. I am proud to say that our department is well-equipped with the right staff and medical resources to deal with complex hematology cases.”

SKMC is a tertiary hospital with a wide spectrum of paediatric services, and is the main Paediatric Hematology service provider in Abu Dhabi, with an inpatient bed capacity of 11 and a 14-bed infusion centre. It follows an integrated multidisciplinary approach in treating a variety of childhood blood disorders through state-of-the-art facilities, cutting-edge equipment, and personalised treatment plans to meet the individual needs of children.

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