The Neurology Department brings hope to children with SMA

From:Children’s Hospital of Soochow University    Release time:2019-12-11

On November 27, 2019, Zhezhe, who suffers from spinal muscular atrophy (SMA), successfully received the intrathecal injection of “Nusinersen” (the world’s first medicine of curing SMA approved by the FDA) in the Neurology Department of our hospital, becoming the first child with SMA in Jiangsu Province to receive the Nusinersen treatment.

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At the age of 13 months, Zhezhe, the patient, gradually regressed his motor functions. He used to be able to crawl, but now he can’t; he was able to sit still, but now he fails to do so; he was able to hold things stably and could hold the feeder with both his hands, but now, it is difficult for him to complete such action. According to Tang Jihong, PhD supervisor and Director of the Neurology Department, Zhezhe is suffering from SMA. As a rare autosomal recessive genetic disease, SMA is one of the 121 rare diseases jointly announced by the five Ministries and Commissions, e.g. the National Health Commission. Though the incidence rate is about 1/6000-1/10000, yet the disease carrier rate is high, that is, one of every 40-50 people carry the disease-causing gene. After the onset of the disease, due to degenerative changes of motor neuron cells in the anterior horn of the spinal cord, children’s body and limb muscles gradually atrophy, thereby losing corresponding motor functions. There will also be multiple system damages such as breathing disorders, swallowing disorders, bone deformation and malnutrition. Hence, SMA has become one of the most common fatal diseases in infants and young children. In severe cases, the patients cannot even live to 2 years old.

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The Neurology Department of our hospital is actively committed to the diagnosis, treatment and management of children with SMA. On April 28, 2019, the Alliance for SMA Centers in China was established in Shanghai. More than 100 experts and scholars specializing in the fields of neurology, rehabilitation and orthopedics at home and abroad gathered to exchange ideas on the status quo and future concerning the treatment of SMA. Our hospital is one of the first batch of 25 member units of the Alliance in China.

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Zhezhe is a lucky patient. In February 2019, the National Medical Products Administration officially approved the use of Nusinersen injection for the treatment of type 5q spinal muscular atrophy (5qSMA), which means that the treatment of SMA in China has stepped into a brand new stage, and this disease is no longer incurable. Upon hearing the encouraging news that Nusinersen was available in China, the family members of Zhezhe brought him to our Neurology Department, hoping to   get treatment with this medicine. Assisted by the SMA Patients Assistance Project of the China Primary Health Care Foundation, and with the support of the hospital leadership, the SMA Diagnosis and Treatment Team of our Neurology Department started to prepare the treatment of children with Nusinersen injection.

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Before the intrathecal injection, the SMA Diagnosis and Treatment Team of our Neurology Department performed a fully individualized evaluation of the child. Huo Hongliang, Deputy Chief Physician of the Rehabilitation Department improved the assessment of the child’s motor development scale; Wu Jiawei (Chief Physician) and Qian Qiu (Deputy Chief Physician) from the Anesthesia Department evaluated and guided the painless lumbar intrathecal injection protocol. With sufficient preparations for the medication, at 8 a.m. on November 27, guided by Chief Physician Tang Jihong and assisted by Attending Physician Shi Xiaoyan, Zhang Bingbing, a skilled neurologist with rich experience in lumbar puncture, smoothly performed intrathecal injection of Nusinersen on Zhezhe. The operation was a success. This is the first case of Nusinersen intrathecal injection carried out in Jiangsu Province. With stable breathing and no adverse reactions, the child was expected to leave hospital on the next day.

For years, the Neurology Department of our hospital has committed to the diagnosis and treatment of difficult and rare pediatric neurological diseases, and has always advocated standardized care, actively carried out new technologies and new projects, so as to better serve the patients. As Nusinersen is currently available in China, the Neurology Department of our hospital is actively carrying out the diagnosis and treatment of children with rare SMA. More patients will be cured in the future. We sincerely hope to constantly bring new opportunities for patients suffering from SMA.