Leukemia is a common malignant tumor, and the incidence of leukemia in various regions of China ranks sixth among all kinds of tumors. Perhaps many Chinese people's earliest understanding of leukemia comes from the popular Korean drama "Blue Life and Death Love", and Eun Hee played by Qiao Mei was killed by leukemia in the mood for love, leukemia is a terrible incurable disease, which has long formed a deep imprint on everyone. However, the chronic myelogenous leukemia we are going to talk about today can be said to be one of the more "lucky" leukemias in the leukemia "family".
Stills from "Blue Life and Death Love".
"Slow granule" is the abbreviation of chronic myeloid leukemia, which is caused by gene mutations caused by translocation of chromosomes 9 and 22 in the patient's body, so September 22 of each year is designated as the International Slow Granule Day. The annual incidence of "slow granules" is about 5 per million, accounting for 1 fifth of all leukemias in China.
Compared with the danger of acute leukemia, "slow grain" does not need to look around for a matching transplant, but can control the disease through oral pills like diabetes and hypertension. Patients with chronic granules can live and work normally, get married, have children, and even engage in their favorite sports. If it is well maintained, this state can be maintained for more than ten years or even longer.
Connect with experts. The story in the film takes place in China more than ten years ago, when the medical level and medical system are far from today's, and the hope and despair of patients with slow grains deeply tug at the hearts of every moviegoer. So, what is the current living situation and medical security of patients with chronic granules?The senior sister interviewed Wang Yanying, the attending physician of the Department of Hematology and Oncology of Beijing Tsinghua Changgung Hospital affiliated to Tsinghua University, and she will give us more professional answers.
Senior Sister: In the movie, we see that patients with chronic granules can actually live and work normally under the control of drugs, even heavy physical labor such as carrying boxes. Will this have an impact on their condition?
Dr. Wang: Generally speaking, patients with chronic granules can live and work normally and will not have an impact on their condition if they use drugs** to achieve a complete hematologic response (i.e., the blood routine returns to the normal range, there are no abnormal naïve cells in the peripheral blood, the symptoms such as abdominal distension and fatigue disappear at the time of illness, and the spleen retracts to normal).However, heavy physical work should be avoided as much as possible. This is because rash, edema, headache, and various gastrointestinal reactions such as nausea, vomiting, and diarrhea may occur with GleevecIn addition, the use of Gleevec will also cause hematologic toxicity such as leukopenia, thrombocytopenia, and anemia, and if you participate in heavy physical labor at this time, it will increase the chance of infection and bleeding.
Senior sister: Can you give us a science popularization of the treatment principle of Gleevec (called Glenin in the movie)?Why is this drug so expensive?Are there legal alternatives in the country now?
Dr. Wang: The pathogenesis of chronic granules is well understood, which is due to the translocation of the ABL gene on chromosome 9 and the BCR gene on chromosome 22 to produce the BCR ABL fusion gene, which causes the abnormal activation of tyrosine kinase, and leads to abnormal cell proliferation, inhibition of apoptosis, and finally cell malignant transformation through downstream signaling pathways. Gleevec, imatinib mesylate, is a tyrosinase inhibitor (TKI) developed by Novartis, which is a targeted drug for BCR ABL fusion genes, which inhibits the function of this fusion gene in tumor cells, and ultimately leads to apoptosis of tumor cells. The high cost of this drug is due to the large amount of R&D investment in the early stage and the protection of the patent term. At present, there are legal generic drugs in China, namely Xinwei, Ganico, and Nolinin.
Senior sister: How can we avoid or delay the disease from entering the blast crisis?
Dr. Wang: Current studies have found that chromosomal aberrations, gene deletions, insertions, point mutations, and mutations in the BCR ABL kinase region are the main mechanisms that cause patients with chronic granules to enter the accelerated phase and blast phase in chronic patients. All patients with chronic granules should use a full dose and full course of TKI according to the doctor's recommendation to avoid interruption or reduction of the doseIn addition, regular monitoring of the condition is very important, and it must be followed up every three to six months according to the doctor's requirements, including blood routine, blood smear, bone marrow, especially peripheral blood fusion gene quantification. In addition, the use of other adjuvant drugs should be avoided as much as possible, and the interaction between other drugs and TKI should be reduced as much as possible, so as to avoid the change in efficacy caused by the interaction.
Senior sister: If you enter the blast crisis period, will the drug lose its effect?Is there a way to save lives?
Dr. Wang: Entering the emergency period, it is not impossible, there are still a variety of options. First of all, the detection of BCR ABL kinase region mutations should be improved, and different regimens should be selected according to the mutation situation, the patient's previous history and underlying diseases. For example, TKI monotherapy or combination chemotherapy may be used to improve remission rates, and allogeneic hematopoietic stem cell transplantation may be performed as soon as possible after remissionOr you can choose to participate in a clinical trial.
Senior Sister: How much does it cost for a year of drug maintenance and routine check-up for a chronic granule patient?
Dr. Wang: The cost of chronic granules is composed of drugs and examinations, and the following expenses are all before reimbursement. If you take the original drug Gleevec, it is about 280,000 yuan;If you are eligible, you can participate in the China Charity Federation Gleevec Patient Assistance Program, which is "buy three get nine free", at an annual cost of about 720,000 RMB;If you take domestic generic drugs, the annual drug cost is about 2-30,000 yuan. The cost of testing is about 5,000-10,000 per year, depending on the condition. At present, most parts of China have included these drugs in the scope of medical insurance.
Senior Sister: What is the current survival rate of patients with slow granules?
Dr. Wang: At present, with the application of TKI drugs, the average survival of patients with chronic granules has been greatly improved, and the 10-year survival rate of patients with the first-generation TKI imatinib has reached 85%-90%, and the average survival time has reached 19 years, or even longerSecond-generation TKI** slow pellets also allow for faster, deeper molecular reactions. If you stick to ** and get tested regularly, most patients can achieve long-term survival.
Senior sister: Is it possible for patients with slow granules to be **?
Dr. Wang: At present, more and more studies have shown that some patients with chronic granules who have achieved a deep molecular response time of more than 2 years with TKI can achieve long-term non-remission, that is, functional. Therefore, a complete cytogenetic response should be achieved as soon as possible, as well as a deeper molecular response, and improving quality of life and functionality is the long-term goal of Slow Granules.
Paying attention to rare diseases requires you, me and him.
This film not only reflects the fact that Chinese patients with chronic granules cannot afford to eat sky-high drugs at the beginning of the 21st century, but also a microcosm of many rare disease patient groups in the world.
Due to the low market demand and high R&D cost of drugs for rare diseases, few pharmaceutical companies carry out R&D and production, and these drugs are called "orphan drugs". These drugs are not only the most expensive, but also not easy to be included in the medical insurance, so they will bring an unbearable financial burden to patients with rare diseases.
Nowadays, the progress of the era of science and technology and the development of social networking have attracted more and more attention from people, such as the "ALS Man" who is known by everyone by the "Ice Bucket Challenge Relay", and the "Peter Pan" who is known by "Orange Smile" and so on. Forces from all walks of life are paying attention to and caring for these rare disease patients, which has attracted widespread attention from the society, and can eventually give them more medical security and social care.