In the life of "lighting and lighting", they want to run like ordinary people

From no medicine to medicine to having medicine

Hope of patients with hemophilia is lit

"You don't know when bleeding will happen, and you are torturing pain every day."

Guan Tao, born in 1971, was diagnosed with hemophilia when he was 7 months old. The pain caused by bleeding accompanied him throughout his childhood. Guan Tao has basically experienced all the bleeding of subcutaneous hemorrhage, joint bleeding, muscle bleeding, visceral bleeding, all the bleeding of hemophilia.

At the age of 9, Guan Tao's right leg was unexpectedly bumped and caused heavy joint bleeding. After that, he could only hold a double -turn activity. However, at the age of 12, Guan Tao finally lost his ability to walk and sat on a wheelchair. He lived carefully all day long, but there was always a step he couldn't cross in front of him.

As a hemorrhagic disease of genetic coagulation dysfunction, the incidence of hemophilia in my country is 2.73/100,000. According to the type of lack of coagulation factors, it is mainly divided into two types of hemophilia A and hemophilia B Essence

A small wound and a teeth at a time will cause the patient to bleed more than bleeding. Patients with severe illnesses will even "spontaneously" bleeding. If they cannot be treated in time, they may lead to disability and even death. According to medical insurance data from the Hematology Hospital of the Chinese Academy of Medical Sciences, the total number of hemophilia patients who need to use drug treatment at present is about 40,000, most of which are men.

Due to the particularity of the symptoms, patients with hemophilia are called "glass people". Once they are sick, they must experience the life of "gently and light".

Warm from holding a group to the outside

In the 1970s, there were no effective drugs in China that could treat hemophilia, and patients could only rely on fresh plasma to save their lives.

Guan Tao broke through the bleeding difficulty of 3 or 4 times a month, and it took 3 to 7 days of recovery each time. In the end, Guan Tao, who had been in the first grade and learned some Chinese Pinyin, had to say goodbye to the campus. In addition to missed the best treatment period, it caused disability, and Guan Tao was difficult to get out of the house and integrate into the society.

Occasionally, Guan Tao met several hemophilia patients. At first sight, physical and psychological trauma could feel the same. After some communication, they had the idea of ​​building a website.

In 2000, the "House of Blood Friends" website was established. This platform connects hemophilia patients across the country together, telling each other and holding the group to warm up, so that people with the same disease have a sense of belonging. Guan Tao said that he had established the "House of Bloods" at the beginning mainly to promote the communication and information release of patients. Later, more and more patients gathered here. Knowledge education.

"The problem of lack of medicine and high treatment cost has always troubled the group of patients with hemophilia." Guan Tao introduced that hemophilia can be treated by injecting coagulation factors, and the amount of coagulation factor is directly linked to the patient's weight, plus the drug's medicine The half -life (the speed of the drug eliminates the speed in the body) is short, and the cost of treatment of hundreds of thousands a year can bear it.

In order to incorporate the treatment costs into the scope of medical insurance reimbursement, Guan Tao has repeatedly submitted a proposal for hemophilia related medical insurance policy. Over the years, Guan Tao and the "House of Blood Friends" have helped many poor patients by raising the funds, and also provided comprehensive assistance in many aspects such as education, skills training and psychological care.

In February 2012, Guan Tao registered and set up a rare illness love center of hemother home at the Beijing Municipal Civil Affairs Bureau. "House of Bloods" was organized by a simple patient to become a private non -corporate unit. As a member of the Capital Charity Public Welfare Organization Federation, the "House of Blood Friends" and the World Hematology Alliance, the China Women's Development Foundation, and Beijing Children's Hospital have formed a good cooperative relationship. ", Will also combine public publicity with major media, so that more people know the existence of this group.

The key to "not disabled" is children

Compared with Guan Tao, Zhong Yu is lucky.

The 15 -year -old Zhong Yu was optimistic and cheerful. Although he had suffered from hemophilia since he was a child, he did not terminate his studies. He could only sit in the classroom whenever a physical education class.

The beginning of everything was a gum bleeding at the age of 1. After a minor bleeding last night, Zhong Yu's mother took him to the hospital to take a blood test. The doctor told her that her son was likely to be hemophilia.

"We don't understand what hemirphful disease is, but how can gums bleed about plasma for plasma treatment." Recalling the treatment process, the two words that Mother Zhong Yu mentioned was "don't understand".

"Do not understand" is the general first reaction of most hemophilia patients and families. Their awareness of the disease often starts from the treatment stage. Even though people obtain diverse information channels, the World Hematology Alliance sets April 17th each year as "World Hematology Disease Day", but in the public impression, hemophilia still looks strange.

The low social cognition of hemophilia, and the lack of timely treatment of treatment, directly leading to the high disability rate and joint malformation rate of patients. When Zhong Yu first found hemophilia, her right arm swelled because of venous blood blood, and she moved to several hospitals to seek treatment, but finally fell into disability due to delaying the best treatment period.

"Treatment of hemophilia is the key to childhood." Professor Wu Runhui, Hematoma Cancer Center, Beijing Children's Hospital Affiliated to the Capital Medical University, said, "The standardized prevention treatment of children is not only aimed at those heavy types. . According to the condition of children's patients, formulating individualized prevention treatment is the optimal treatment method, which can achieve the purpose of not disability and death. "At present, the treatment of hemophilia is mainly the treatment Two types of treatment and prevention. Domestic use is generally treated on demand, that is, after bleeding, input coagulation factors to help patients stop bleeding. The latest "Guidelines and Consensus Guidelines and Consensus of China" pointed out that my country has the basic conditions for the treatment of hemophilia prevention and should actively promote preventive treatment to reduce the disability rate of hemophilia patients in my country and improve the quality of life. Professor Yang Renchi, the leader of the China Intelligence Association and the Director of the Hematology Hospital of the Chinese Academy of Medical Sciences, emphasized that "preventing treatment is not to prevent hemophilia, but refers to hemophilia patients After intracranial hemorrhage, immediately start regularly accepting coagulation factor injection to prevent re -bleeding and joint damage, so as to reduce the residual rate and prevent fatal bleeding again. "

Curring dysfunction (left) and normal coagulation (right) schematic diagram

In 2012, when Zhong Yu's mother intends to let her son prevent treatment, the doctor told her that Zhong Yu's body had inhibitors (antibodies) in her body. The existence of inhibitors means that the effectiveness of coagulation factor treatment is reduced and the risk of bleeding in patients increases.

According to statistics, about 30%of patients with severe hemophilia A will produce inhibitors. Such patients need to infuse a large amount of coagulation factors for inhibitors. The reorganized human coagulation factor ⅶA or coagulantlase original complex was used for hemostasis treatment.

"The emergence of inhibitors has brought greater challenges to patients and families, but it cannot be used to waste food, but it needs more active treatment. The difficulty is that more coagulation factors and economic support." Professor Wu Runhui emphasized that many efficient and innovative innovations The research and development of medicines provides more choices for the treatment of hemophilia. Among them, the restructuring human coagulation factor that has been listed in China for 20 years ⅶA is used to stop hemostatic treatment of patients with inhibitors. In 2017, it was included in the national medical insurance directory and successfully renewed in 2019 and 2021. Treatment difficulties.

Zhong Yu's bleeding after 12 years of bleeding is particularly frequent. At most, twice a week, the left arm blood vessels have been tied with a small nodule. Later, the doctor gave Zhong Yu's body a venous catheter. He had lived with a catheter for three years. He was going to inject the coagulation factors through it every few days, and Mother Zhong Yu had already cooked the light car for dating.

"Seven factor (reorganized human coagulation factor ⅶA) is indeed used, and now we have entered the medical insurance to reduce our economic burden. It's difficult. "Mother Zhong Yu said.

Help more, let them run freely

Since 2000, the diagnosis and treatment of Chinese hemophilia has undergone rapid development. From "no medicine can be medical" to "medicine can be available", the situation of patients is gradually improving. Passion, return to normal study, work and life.

In 2009, the state established an information management system for hemophilia and established the information management center of hemophilia. In 2012, the state proposed the establishment of a hemophilia class diagnosis and treatment system to determine the designated medical institutions of hemophilia, and improve the ability of hemophilia screening and conventional treatment of grass -roots medical and health institutions. At present, the national medical insurance directory incorporates a variety of hemophilia drugs into the scope of national medical insurance, especially the prevention and treatment of children is also included in the scope of medical insurance. At the same time, provinces have introduced corresponding medical insurance reimbursement policies for drugs such as hemophilia and other provinces. Incorporate hemophilia into the medical insurance.

In addition to the gradual improvement of medical insurance coverage, medical insurance institutions in many provinces have also begun to actively explore innovative methods that can be further expanded. For example, in 2022, in the Guangdong Alliance Collection, which participated in the 11 provinces and autonomous regions, the reorganized human coagulation factor in the treatment of type A hemophilia was selected, reducing the economic burden of patients and their families, and it is expected to help more blood. Patients with friendly diseases conduct preventive treatment.

In terms of improving drugs, pharmaceutical companies also play an important role. As early as 1985, Nuo and Nord decided to abandon business interests, support the first drug research for patients with hemophilia and inhibitors, and extend their hands to 3,500 hemophilia patients around the world at the time, ending the accompanying inhibitors, ending the inhibitors The patient has no medicine. To this day, Novo Nord has continued to cultivate the field of healing disease, providing patients with better, more accessible, and easier treatment plans.

In 2018, it was initiated by the "House of Blood Friends", and Nuo and Nord's public welfare supported the "Creation of its Traces-Rare Bleeding Disease Aid Project of Bloody Family". As of 2021, a total of 28.98 million yuan was aid, and 739 patients were rescued.

At the same time, in order to promote the academic exchanges in the field of hemophilia and rare hemorrhagic diseases, and continued to promote the improvement of diagnosis and treatment, Novo Nordon launched the Nuo and Nord Blood Summit Forum in 2019. In addition, through the care of patients with a variety of hemophilia patients, carry out a number of care activities such as disease science popularization, family care, psychological rehabilitation, etc., helping hemophilia patients to improve the cognition of disease, gain more comprehensive disease management knowledge, and call on the whole society to appeal to the whole society Pay attention to and support hemophilia. For the hemophilia group, the past demands were "survive"; the appearance of medical methods and the treatment of drugs, they bid farewell to the painful life; today, national policy, medical progress, and social care of society light up them " The hope of not disabled.

"I have a photo of children with bloody friends in 2018. They chase and make trouble, which is exactly the same as normal children. This is also the goal of our efforts." Professor Wu Runhui said.

Maybe this goal can't take too long. Emerging gene therapy has repeatedly made breakthroughs in clinical studies, or brings "once and for all" solutions to the field of hemophilia. According to Professor Yang Renichi, who participated in the first liver -targeted glands -related virus (AAV) hemophilia B gene therapy project in Asia, "Among the 10 patients with hemophilia, 3 of the earliest enrollment were only administered through intravenous administration. For two years, the level of coagulation factor is maintained at about 30%. Studies have confirmed that gene therapy is effective for hemophilia B for a long time, allowing us to see the possibility of genetic treatment healing hemophilia. "

"I hope that soon, patients with hemophilia can achieve sufficient effective treatment, no pain or no real" healing "." Now, in the eyes of Guan Tao, 51, hemophilia is not so terrible. In June this year, Zhong Yu admitted to the key high school in Tianjin Jinnan District, and ignited the hope of the family.

"If hemophilia can really cure, I want to experience the feeling of exercise. I want to try table tennis, badminton, running and the like." Zhong Yu said.

Hope his dream can be realized as soon as possible.

(The patient Zhong Yu in the text is a pseudonym)

Author: Xiao Wanxuan

Edit: Wang Xiaoyi

Operation editor: Wang Lin

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