Reza Shervin Badv1, S Sharareh Dehghani2, Maryam Behfar2, Batoul Ahadi3, Abdolreza Tabasi4, Amir Ali Hamidieh5. 1. Children's Medical Center, Pediatric Center of Excellence, Tehran University of Medical Sciences, Iran. 2. Hematology, Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Iran. 3. Department of Biostatics, Para-Medical Faculty, Shahid Beheshti University of Medical Sciences, Iran. 4. Department of Neuro-ophthalmology, Tehran University of Medical Sciences, Iran. 5. Children's Medical Center, Pediatric Center of Excellence, Tehran University of Medical Sciences, Iran; Hematology, Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Iran. Electronic address: aahamidieh@sina.tums.ac.ir.
Abstract
OBJECTIVES: Malignant Infantile Osteopetrosis (MIOP) is a rare inherited disorder with neurological complications, notably visual impairment and decrease of hearing level. Although Hematopoietic Stem Cell Transplantation (HSCT) has been approved as the only curative treatment for these patients, the exact impact of it on visual and hearing level is still unclear. STUDY DESIGN: We analyzed the P2 latency and amplitude from Visual Evoked Potentials (VEP) of 10 patients (20 eyes) and the threshold of wave V from Auditory Brainstem Response (ABR) of 15 patients (30 ears) with MIOP before, 6 and 12 months after HSCT. RESULTS: Before the HSCT, 10/30 ears demonstrated some degree of hearing loss; while only 3/20 eyes had P2 wave latencies in normal range for age. Using GEE models, it was shown that 12 months after HSCT, wave V threshold of ABR of the patients was significantly lower compared to its value from before the transplant (p value: 0.04). The analysis of latency and amplitude of P2 wave of VEPs showed no significant difference between before and after the transplant. CONCLUSION: This study clearly showed that HSCT can improve the hearing level of the patients in terms of ABR threshold. Although HSCT made no significant improvement in latency of P2 in VEP of the patients, it can be concluded that transplant can halt visual regression in these patients. Early diagnosis of MIOP with this objective tools and subsequently early HSCT in these patients can decrease the rate of neurological complications of MIOP and improve the quality of life in them.
OBJECTIVES:Malignant Infantile Osteopetrosis (MIOP) is a rare inherited disorder with neurological complications, notably visual impairment and decrease of hearing level. Although Hematopoietic Stem Cell Transplantation (HSCT) has been approved as the only curative treatment for these patients, the exact impact of it on visual and hearing level is still unclear. STUDY DESIGN: We analyzed the P2 latency and amplitude from Visual Evoked Potentials (VEP) of 10 patients (20 eyes) and the threshold of wave V from Auditory Brainstem Response (ABR) of 15 patients (30 ears) with MIOP before, 6 and 12 months after HSCT. RESULTS: Before the HSCT, 10/30 ears demonstrated some degree of hearing loss; while only 3/20 eyes had P2 wave latencies in normal range for age. Using GEE models, it was shown that 12 months after HSCT, wave V threshold of ABR of the patients was significantly lower compared to its value from before the transplant (p value: 0.04). The analysis of latency and amplitude of P2 wave of VEPs showed no significant difference between before and after the transplant. CONCLUSION: This study clearly showed that HSCT can improve the hearing level of the patients in terms of ABR threshold. Although HSCT made no significant improvement in latency of P2 in VEP of the patients, it can be concluded that transplant can halt visual regression in these patients. Early diagnosis of MIOP with this objective tools and subsequently early HSCT in these patients can decrease the rate of neurological complications of MIOP and improve the quality of life in them.