Source of this article: Times Finance Author: Zhang Yuqi
At the time of his first surgery in August 2020, Ash had just turned one year old when his intestine was cut off about 40 centimeters. At this time, the normal length of the intestine of a child of the same age is more than 2 meters. Today, the 4-and-a-half-year-old Chi intestine is getting shorter and shorter, with only 30 centimeters left in the small intestine and 20 centimeters in the distal colon. As soon as you lie flat, what you eat in your stomach will rush up with gastric juice, and then vomit out a horribly colored liquid. If you don't pay attention to eating, you will have severe diarrhea and even dehydration.
Like many children, Ash is full of curiosity about food, and will cry against it if he doesn't give it something delicious. But repeated diarrhea and never-ending injections changed his imagination of the world. Ignorant, he gradually realized one thing - "If I eat too much or eat something bad, I will vomit very badly."
This is a daily portrait of a patient with short bowel syndrome (SBS). The length of the intestine is measured in centimeters, and it is limited to eating or even unable to eat, and it is necessary to carry a nasogastric tube and an indwelling needle with him, and even need to inject enteral nutrition and parenteral nutrition for life. A number of patients with short bowel syndrome and their families told Times Finance that this is the norm in their lives.
Patients with short bowel syndrome need to carry a nasogastric tube and an indwelling needle with them to meet the needs of parenteral nutrition infusion. Image source: Courtesy of interviewee Song Li
According to the Rare Disease Information Network, short bowel syndrome refers to the syndrome of diarrhea, acid-alkaline/water/electrolyte disorders, and nutrient absorption and metabolic dysfunction after extensive small bowel resection or absenteeism caused by various reasons. The small intestine of a normal adult is about 3 meters ~ 8.5 meters, while the length of the small intestine of patients with short bowel syndrome is often less than 2 meters or even less than 1 meter. It is estimated that the prevalence of adults in mainland China is about 0.73 per 1,000,000, and the incidence trend is increasing year by year.
Wang Jian, director of the Department of General Surgery at the Fourth Affiliated Hospital of Nanjing Medical University, told Times Finance that most rare diseases are hereditary, but short bowel syndrome has its particularities. Congenital short bowel syndrome is very rare, and most patients have to remove part of the bowel due to intestinal necrosis, Crohn's disease, radiation enteritis and other diseases caused by mesenteric vascular disease or volvulus in childhood or adulthood, or repeated abdominal surgery, and the remaining small intestine is too short to form a short bowel. Some people with short intestines can also maintain their own intestinal functions through residual intestinal compensation, but for other groups with short intestines, the residual intestines are absolutely insufficient and cannot meet the needs of the body through oral nutrition, which will eventually lead to the dilemma of "intestinal failure", and long-term enteral and parenteral nutrition support is needed to maintain survival and growth and development.
Earlier this year, Takeda Pharmaceutical's product Teduglutide for Injection (trade name: Ruiweishu) was officially approved by the National Medical Products Administration (NMPA) for the treatment of adults and children aged 1 year and older with short bowel syndrome. This is the first human glucagon-like peptide-2 (GLP-2) analogue for the treatment of short bowel syndrome in China, and its approval not only brings the ultra-rare disease group of "short bowel people" into the public eye, but also gives them new hope for treatment.
But not every "short-hearted person" can use or afford innovative drugs. The final pricing of tedoliglutide has not yet been announced, but Zhou Hui, a family member of the patient, told Times Finance, "If the child uses enteral nutrition and parenteral nutrition therapy, the annual cost may exceed 200,000 yuan, but if the innovative drug teduglutide is used, the annual cost may exceed 1 million yuan." ”
They are still trapped in disease. Faced with the million-dollar drug price, patients and their families are discouraged and swayed by the unknown treatment effect. No one could tell them the exact solution.
"It took us more than two years to take the step of returning home"
On March 6, 2023, Song Li and Xiaozhi finally took the first step back home. At this time, it has been two years and six months since they were first hospitalized at the Children's Hospital of Zhejiang University School of Medicine.
Ash is a child with a very rare short bowel syndrome. According to the 2023 Survey Report on the Diagnosis and Treatment Status and Disease Burden of Patients with Short Bowel Syndrome in China released by the Cord Center, the prevalence rate in the global population is between 0.3-4 per 100,000 (United States) and 0.1-4 per 100,000, and only a very small number of people suffer from this disease.
Ash was born in August 2019, and before his first birthday, he lived a happy time like every ordinary toddler and could eat anything he could eat at his age. But it all came to an abrupt end on the morning of August 30, 2020. Ash underwent five laparotomy surgeries for volvulus, and finally his small intestine was only 30 centimeters left, and his distal colon was only 20 centimeters, and he was diagnosed with short bowel syndrome.
The doctor told Song Li that Ash's remaining intestines were like rags and were not functioning properly, and that he could try to eat and enteral nutrition, but his absorption was extremely limited. Since then, parenteral nutrition support has become a necessity in Ash's life.
"During the more than two years I was hospitalized, I never ate in front of him, and only when he fell asleep did I dare to eat secretly. Song Li told Times Finance.
In order to treat their son's illness, Song Li's family traveled to many hospitals for medical treatment. In the fall of 2020, Xiaozhi came to the Children's Hospital of Zhejiang University School of Medicine for treatment, and the whole family moved from their hometown in Guangdong to Hangzhou. Song Li resigned to become a full-time mother and lived in the ward with Xiaozhi, and the clothes of the mother and son were stored in the bedside table less than one meter high next to the bed. As spring turned to autumn, other children were discharged from the hospital one after another, and they were not able to get out of the hospital.
"We are one of the longest-standing and most severe patients with short bowel syndrome, and even in the elevator, the doctors and nurses can recognize Ash, and the department treats his illness as a typical case. Song Li teased.
At first, Song Li and her husband only wanted to save Ash's life, no matter what method they used. Later, the person was saved, but the intestines became shorter, and he was hospitalized for a long time, and his quality of life continued to decline after being infused with nutrient solution.
After learning that a child had left the hospital, the parents began to use their professional knowledge to provide home care for the child, and Song Li had an idea - to take Xiaozhi out of the hospital.
"I need to take him home. Song Li said, "The later you leave the hospital and the later you return to your family and society, the greater the gap between Ash and the outside world, and the more difficult it will be for him to integrate into daily life." Maybe it's still young, but later he slowly discovers that he's different, and it's a psychological shadow for a lifetime. Going home early may improve the situation, but he is still ignorant, and others may just ask. ”
Song Li quickly took action. After obtaining the doctor's discharge opinion, she was learning nursing knowledge every night, how to make intravenous nutrition, how to dispense medicine, and constantly building up the psychology of herself and her family.
"I asked the doctors and nurses if I didn't understand, and they were also talking about professional nursing knowledge in a language that I could understand, and with their help, Ash's discharge from the hospital and family therapy after discharge became smoother. Song Li sighed.
Home care, a protracted battle
It's not easy to go home, and it's even harder to go home.
According to the Chinese Consensus on the Diagnosis and Treatment of Short Bowel Syndrome, there are two major treatment directions for short bowel syndrome: intestinal rehabilitation and intestinal replacement, including nutritional support, medical treatment of bowel rehabilitation to promote adaptive compensation of residual small intestine, non-transplant surgery and small intestine transplantation. In terms of nutritional support, different forms of nutritional support are given according to the different degrees of adverse manifestations of patients with short bowel syndrome. According to the statistics of the Cord Center for Rare Diseases in 2023, among the 108 patients who participated in the questionnaire, 60% of the patients still rely on parenteral nutrition support therapy (PN) for a long time, and only 40% of patients are out of PN treatment and only need enteral nutrition support and diet to maintain nutritional levels. Adolescent patients, in particular, are generally more dependent on parenteral nutrition support.
Among the four patients interviewed by Times Finance, they were all treated with nutritional support, but the degree of treatment was different due to the difference in individual diseases. Patients with mild disease require only oral nutrition solutions and are able to eat, while those with severe disease need to be given enteral nutrition with a nasogastric tube for 24 hours and intravenous parenteral nutrition. Although long-term nutritional support can sustain a patient's life, it can seriously affect the patient's quality of life and daily life.
In terms of physical health, due to the disease itself, patients tend to have long-term diarrhea, and due to insufficient nutrition, patients are often lower in height and weight than their peers. Children with more serious conditions like Ash have not only been hospitalized for a long time in the past, but even if they meet the discharge criteria, they still face many challenges in their quality of life after returning home, even more so than when they were hospitalized.
Wang Jian explained to Times Finance that parenteral nutrition is a special treatment, which requires a daily mix of carbohydrates, fats, amino acids, electrolytes, trace elements, vitamins, water and other nutrients into a nutrition bag for continuous infusion, which often needs to be achieved in the hospital. If it is configured at home, it is necessary to solve the problem of medical insurance reimbursement, and second, to provide adequate training to the patient's caregiver and be able to obtain guidance from medical personnel at any time.
Home dispensing operations. Image source: Courtesy of interviewee Song Li
"Parenteral nutrition solution requires special central venous catheter infusion, commonly used tubing includes PICC (peripheral venous puncture central venous catheterization), CVC (central venous catheter), infusion port, etc., this pipeline system is prone to infection, long-term use is also easy to cause blockage and even thrombosis, resulting in the loss of precious venous access. These are fatal complications for these patients, and in order to prevent them adequately, very high demands are placed on the care process. Wang Jian pointed out.
The occurrence of these complications means that after entering the home care model, caregivers must not only learn to dispense and care for patients, but also establish a sterile environment in the residence to reduce the risk of infection.
Song Li told Times Finance that in the process of Xiaozhi's repeated search for blood vessels to indwell PICC tubes, she already knew the seriousness of the infection. From the first hospitalization to the present, Ash's PICC tube has been replaced 8 times, and the place where the tube used to be left will be red and allergic, there is no place to place the tube in the arm, and the neck can be placed but it is difficult to care, so at present, it can only be considered to put the tube on the leg. "We didn't know how to care in the early days, but now we have realized that we must protect the catheter. ”
After being discharged from the hospital, Song Li opened up a space in their 50-square-meter home as an operating table for Xiaozhi's treatment drugs. It's a sterile area that's set up and she has to disinfect and wipe it down every day. In her hometown in the countryside, even though they only went back once for the Chinese New Year, she also set up a separate sterile room, equipped with ultraviolet lamps, medicine dispensing carts, protective clothing, etc.
A sterile table for home preparation of medicines. Image source: Courtesy of interviewee Song Li
Wang Jian pointed out to Times Finance that the problems faced by patients with short bowel syndrome are not only a series of problems caused by the disease itself, but also complications caused by these systematic treatment measures. "Especially after the return to home care, the current situation in China is that such care is in a disorderly state, the professionalism of caregivers is often seriously insufficient, home parenteral nutrition is not widely practiced in China, and the limited individual experience is worrying. Wang Jian said.
Song Li has a record book, which records in detail the details of Xiaozhi's daily discharge/amount, milk volume, supplementary food, urine and urine, nursing, and the time is also accurate to the second. Song Li told Times Finance that these details are what she observes on a daily basis, and any small change in the value will have a great impact on the child.
A breakdown of the child's daily discharge volume. Image source: Courtesy of interviewee Song Li
It is precisely because of this that any slight progress in Ash's life is even more precious.
After about half a year of home care, Ash's intravenous nutrition therapy was reduced from 16 hours to 13 hours a day. The shortening of 3 hours is a huge step forward for this protracted treatment.
After being discharged from the hospital, Ash's mood has changed dramatically, he is full of curiosity about the world again, and his language skills have also improved by leaps and bounds. Song Li told Times Finance that these changes in Xiaozhi made the whole family sincerely happy, "the child is getting better".
Innovative drugs are coming, and patients are trapped in millions of costs
"I often joke with my kids that you're the hidden part of the blind box. Han Bing, a family member of a child with short bowel syndrome, told Times Finance that when the children in the family mentioned the disease to her, she would answer like this.
Short bowel syndrome and even rare diseases are such "hidden items" that can be drawn by accident.
After the lottery, not being able to eat properly is only the first hurdle they have to face, and then there are more serious problems such as medical treatment, treatment, money and education. It's just that these problems exist in the present, but the solutions are far away.
In particular, in terms of treatment, until the launch of Takeda Pharmaceutical's teduglutide in February this year, there was no innovative drug in China that could treat patients with short bowel syndrome. However, the launch of this drug does not mean that patients with short bowel syndrome have a cure, and the quality of life problem is solved.
Patients are stuck on price in the first place. "When using enteral and parenteral nutrition, I roughly estimated the cost, 3,000 yuan a month, about 40,000 yuan a year, plus some hospitalization miscellaneous expenses, the annual cost is about 50,000~60,000 yuan. Han Bing told Times Finance that in Beijing, both of them go to work and can still support this expense. However, the cost of innovative drugs exceeds one million yuan a year, and it is impossible for their families to buy drugs at their own expense, unless they enter the medical insurance, there is hope.
In September last year, short bowel syndrome was officially included in the "Second Batch of Rare Disease List". Up to now, more than 80 drugs for the treatment of rare diseases have been included in the list of drugs in the National Medical Insurance Drug List. However, as a newly marketed drug, it still takes time for teduglutide to be on the medical insurance negotiation table, and whether it can finally enter the medical insurance depends on the game situation of all parties.
A number of interviewed patients' families also told Times Finance that new drugs are not only a problem for them to not be able to use, but also a problem of whether they can be used, not every patient is suitable for innovative drugs, with innovative drugs, how much can parenteral and extraintestinal support nutrition be reduced, if they cannot be reduced, there is no way to improve the quality of life.
"We went to inquire about this innovative drug, and the doctor told us that Ash has short intestines, and if we have money, we can try it. Song Li told Times Finance that they evaluated the effect and price, but the evaluation results did not meet expectations, so they could only temporarily give up the use.
However, in Wang Jian's view, the domestic listing of tendoliglutide is inspiring. "Especially in adult short bowel disease, there is currently limited space for intestinal rehabilitation through nutrition, surgical plastic surgery and other means, and there is an urgent need for a breakthrough in the field of drug treatment to break this silence. Wang Jian said to Times Finance.
Good news followed. In April this year, Huixiabao (2024 Edition) and Shanghai Huibao (2024 Edition) successively included ticoliglutide in the domestic specific drug list, which means that the drug burden of patients is expected to be greatly reduced.
The person in charge of the "Bowel Health Care SBS Patient Care Center", an organization for patients with short bowel syndrome, told Times Finance that the listing of teloliglutide has brought hope to patients, and because the drug has not been included in the medical insurance for the time being, local patients are looking forward to it, hoping that the local Huimin Insurance can include teloliglutide, so that more patients have hope of using it. The person in charge also called for all sectors of society to work together to make patients have medicine and affordable treatment in the form of multi-level guarantees such as Huimin Insurance and charitable assistance.
(At the request of the interviewee, Zhou Hui, Song Li, and Han Bing are pseudonyms in the article)