World Rare Disease Day | Primary myelofibrosis: a rare disease that often affects the elderly

Not all rare diseases occur in childhood. Primary myelofibrosis (PMF) is a clonal myeloproliferative disease caused by abnormal hematopoietic stem cells. It often occurs in the elderly, with an onset age of 50 to 70 years old. At the same time, as the disease progresses, it may lead to heart failure, vascular complications, etc. in the late stage, and even progress to leukemia, endangering the patient's life.

1. Identification of the disease - Spleen enlargement and distension, abnormal phenomena

Clinically, the symptoms of PMF are mainly manifested in spleen size, blood cell count and some physical changes. These abnormal signals are warnings from the body. If similar symptoms occur, we should seek medical attention in time to confirm the health status of the body earlier and receive symptomatic treatment as soon as possible.

2. Disease diagnosis - blood tumors, 7 tests

When PMF is suspected, patients can visit the outpatient department of hematology for timely screening. At present, the laboratory tests that must be performed on patients suspected of PMF mainly include the following: peripheral blood cell count; bone marrow puncture smear and peripheral blood smear classification count; bone marrow biopsy biopsy pathological cytological analysis; chromosome karyotype analysis; biochemical examination (serum erythropoietin level, serum iron, etc.); ultrasound or CT examination of the liver and spleen, and MRI is recommended to measure the patient's spleen volume; patients who may receive hematopoietic stem cell transplantation also need HLA matching.

3. Treatment: Combining Chinese and Western medicine to relieve illness and prolong life

1. Western medicine treatment: Due to the complexity of the disease's own symptoms, the clinical treatment of PMF involves multiple aspects. Different clinical symptoms will have different therapeutic drugs and treatment methods. At present, Western medicine treatment is mainly palliative treatment, targeting the clinical symptoms of PMF. In addition, according to the degree of disease risk, allogeneic stem cell transplantation will be considered after weighing the pros and cons.

2. Treatment with Traditional Chinese Medicine: From the perspective of Traditional Chinese Medicine, PMF belongs to the categories of "deficiency", "symptoms", "accumulation" and "blood syndrome". For deficiency disease, Zhang Zhongjing's "Golden Chamber Synopsis·Deficiency Disease" includes Xiao Jianzhong Decoction, Huangqi Jianzhong Decoction, Bawei Shenqi Pills, Shudi Pills, Dahuang Zhechong Pills and other prescriptions for deficiency disease, all of which are mainly sweet and warm tonic. Modern doctors mostly follow the ancient method, and the treatment of PMF is mainly to promote blood circulation and remove blood stasis, strengthen the body and replenish deficiency, or both. The methods of replenishing deficiency and promoting blood circulation and removing blood stasis will almost run through the entire treatment process.

IV. Nursing - Reasonable diet and health protection

For PMF patients, it is recommended to supplement with rich high-quality protein every day, which can improve the body's anti-cancer ability and promote bone recovery. During the treatment of PMF, the sugar metabolism cycle in the patient's body is often disrupted. Therefore, PMF patients should pay attention to timely supplementing foods with high sugar content to prevent the body's glycogen content from being too low, affecting the body's recovery process. In addition, some anti-cancer foods can also be eaten. Fried, fishy and irritating foods will affect the body's absorption of calcium, phosphorus and other nutrients, which is not conducive to the cure of myelofibrosis. Therefore, PMF patients are advised not to eat this type of food as much as possible.

(Author: Wang Junnan and Liu Haiting, Department of Pharmacy, Dongzhimen Hospital, Beijing University of Chinese Medicine)