Hoda Al Taniji (Abu Dhabi)
The Abu Dhabi Stem Cell Center succeeded in performing a bone marrow transplant on a girl who was diagnosed with Krabbe disease, a rare life-threatening genetic disorder, in which the affected child only survives for 5 or 6 years, during which the child’s nervous system deteriorates. This achievement, the first of its kind in the country, reflects the commitment of the Abu Dhabi Stem Cell Center to find modern and advanced treatments for rare diseases, and to advance the wheel of innovation and medical progress at the level of the country and the region.
The rare hereditary Krabbe disease results from a deficiency in the production of myelin, which is the protective covering of nerves in the brain. This deficiency leads to severe nerve damage and disruption of the nervous system. Urgent medical intervention is necessary before the child reaches three months of age to prevent major damage to the nerves. The brain can threaten the child’s life, which reflects the importance of early detection and diagnosis of the disease.
A bone marrow transplant was performed on the newborn before she turned three months urgently. After searching for a donor from her relatives, it was found that she was genetically identical to her grandmother, but the grandmother was pregnant at the time, and it is not recommended to donate during pregnancy. Fortunately, the grandmother was in the eighth month of pregnancy. Because of her perfect match with the baby girl and the need to quickly carry out the transplant, the grandmother was delivered “by Caesarean section” at the beginning of the ninth month, in cooperation with the Corniche Hospital, where the Abu Dhabi Stem Cell Center team performed a bone marrow withdrawal procedure, during the birth process, and under the same anesthesia from Without any side effects on the health of the donor grandmother and the newborn.
This case demonstrates the important role of early diagnosis and intervention in the treatment of Krabbe disease, as 90% of cases appear between the ages of one month and seven months of age.
Accordingly, Dr. Mansi Sachdev, consultant in pediatric hematology, oncology and bone marrow transplantation, and Dr. David Dennison, consultant in hematology and bone marrow transplantation, in cooperation with Dr. Salima Wani, consultant in obstetrics and gynecology at Corniche Hospital, and the medical team, collected stem cells from the grandmother. During the birth process. The child was also prepared for a bone marrow transplant for a few days, and then the donated stem cells were transferred to her at the Abu Dhabi Stem Cell Center.
For her part, Dr. Sachdev said: “The bone marrow transplantation process for the girl represents a medical achievement in our battle against hereditary diseases, and it also confirms our ability to provide precise and advanced care here in Abu Dhabi through the Abu Dhabi Bone Marrow Transplantation Program at the Abu Dhabi Stem Cell Centre, which includes an elite group of Doctors of hematology and bone marrow transplantation for children and adults, equipped with the latest laboratories and medical devices.
She added: We are a center of excellence in hematopoietic stem cell transplantation accredited by the Abu Dhabi Department of Health, and we believe in the importance of cooperation between doctors and various medical institutions, which enables us to provide comprehensive care for patients, and this is what we provided to the girl, as we succeeded in providing her with the necessary treatment in a timely manner. This is in cooperation with Corniche Hospital.
In turn, Dr. Dennison highlighted the important results of this operation, saying: “After the transplant, the enzyme levels, which were close to zero, became within the normal range for the child. “With the enzyme still at normal levels, bone marrow transplantation represents a cure for her disease, and it is the only cure for Krabbe disease.”
Dr. Salima Wani said: “The cooperation in treating the little girl between the Corniche Hospital and the Abu Dhabi Stem Cell Center reflects the extent of our commitment to applying the highest standards of patient safety, obtaining the necessary support, and putting their health as a priority. The success of the treatment indicates the importance of such cooperation between health institutions to provide comprehensive care.” For patients.
Bone marrow transplantation offers promising results in the future for patients suffering from this disease and other genetic diseases. The efforts made by the Abu Dhabi Stem Cell Center contribute to expanding the horizons of treating various genetic disorders, which is in line with the center’s commitment to providing innovative curative treatments.
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