Primary myelofibrosis (PMF) is a type of myeloproliferative tumor (MPN), which is a progressive malignant hematological disease. About half of the patients died due to the progression of the disease. Without effective treatment, the patient’s symptoms continue to get worse and the quality of life continues to deteriorate. Rucotinib is a targeted inhibitor of JAK pathway, which meets various therapeutic purposes such as shrinking the spleen, improving symptoms, prolonging survival and improving bone marrow pathology. This article shares a case of PMF patients with cardiac insufficiency who obtained clinical benefit after first-line use of rocotinib to provide clinical reference.
No.1
Case brief introduction
Author introduction
Lu Wei
First Affiliated Hospital of Nanchang University
1. Medical history brief introduction
male patient, 69 years old. Seeing a doctor because of "recurrent chest tightness and asthma for 1 year, worsened by 1 month." Physical examination: appearance of anemia, splenomegaly, 8 cm below the ribs.
auxiliary examination:
blood routine : WBC 3.91 ×109/L, PLT 281 ×109/L, Hb 50 g/L, N 82.5%. 0% of nucleated red blood cells and 35.2% of large platelets.
LDH 447 U/L, BNP 4750.08 ng/ml.
Chest and abdomen CT: localized emphysema, scattered exudation in both lungs, a little pleural effusion on both sides; enlarged heart; decreased heart cavity density, suggesting anemia; pericardial effusion; Kidney cyst may be.
Holter electrocardiogram : 1. The whole process is atrial flutter with fast ventricular rate, the minimum ventricular rate is 66 beats/min; 2. T wave changes can be seen during the monitoring process; 3. Incomplete right bundle branch block.
Cardiac ultrasound : large double room, large right ventricle, mild to moderate pulmonary hypertension, pericardial effusion, ejection fraction 60%.
Bone marrow cell morphology : Bone marrow hyperplasia is reduced, no small grains, oil droplets are seen, granular lobed nucleus> rod-shaped nucleus, mature red blood cells are seen in teardrop shape.
Bone marrow cell flow cytometry : The proportion of myeloid blasts is not high, mainly in myelomyelocytic cells and later stages. No abnormal expression of differentiation antigens are seen, some cells express CD56, and the proportion of basophils increases.
Bone marrow biopsy : Fibrous tissue is extensively proliferated, granular red cells are scattered or focally distributed, mainly in the mature stage, megakaryocytes increase, scattered in clusters, some cells are less lobed, and a few lymphocytes are scattered.
Reticulated fiber dyeing (MF-2) . Immunohistochemistry: CD34 individual+, CD117 small amount+, MPO granulocyte+, Lysozyme fraction+, CD42b megakaryocyte+, CD20 small amount+, CD56 individual+, CD138 plasma cell+, CD30-.
gene : JAK2-V617F positive, 86.73%. MPL and CALR genes were negative, and myeloid genes were negative.
MPN-10 Score : 37 points.
2. Admission diagnosis
1, primary bone marrow fibrosis (IPSS score 3 points, DIPSS score 4 points) intermediate risk-2
2, pulmonary hypertension
3, cardiac insufficiency
4, liver cysts
5, history of renal cysts
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1zz3zz1 After diagnosis and treatment, Rucotinib was given 20 mg bid by
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2. Treatment outcome
patient's hemoglobin gradually increased, the platelet count was stable, the spleen was significantly reduced, and the MPN-10 score dropped to 10.
So, reviewing the cases, the patient with primary myelofibrosis with pulmonary hypertension and cardiac insufficiency, how to choose the best plan? What are the excellent effects of Rucotinib in treating myelofibrosis? Listen to what the experts say.
No.3
Let me talk about
expert introduction
Wang Zhi
Chief physician of the Department of Hematology, Wuxi Second Hospital, Nanjing Medical University,
[Wang Zhi said case]
This case is a typical primary myelofibrosis patient with pulmonary hypertension, cardiac insufficiency, etc. . Patients with primary myelofibrosis have poor quality of life and short survival. The JAK2 inhibitor can prolong the survival of patients, effectively improve the quality of life of patients, improve various physical symptoms in MPN-10, and shrink the spleen. Among patients who can be transplanted, the combined use of JAK2 inhibitors can also further improve the transplantation efficacy of primary myelofibrosis. The relationship between patients with pulmonary hypertension and bone marrow fibrosis can be further explored. Rucotinib has a very important position in the current treatment of primary myelofibrosis, and its indications have been further expanded, such as GVHD, acute promyelocytic leukemia with differentiation syndrome, and high inflammatory factor status. Some patients are diagnosed when they enter the stage of hematopoietic failure. There are often obvious anemia and thrombocytopenia. Especially thrombocytopenia restricts the use of rucotinib. It is necessary to fully communicate with the clinician and the patient. The dose is gradually increased cautiously, so that the patient can obtain clinical benefits such as physical symptoms and contraction of the spleen.
Expert introduction
Song Qiang
Deputy Chief Physician of the Department of Hematology, Qilu Hospital of Shandong University
[Song Qiang said case]
The diagnosis of this patient is very clear primary myelofibrosis, clinical symptoms: anemia, splenomegaly, bone marrow pathological fibers At 2°C, teardrop-like red blood cells can be seen in peripheral blood, lactate dehydrogenase is increased, and JAK2-V617F is positive. After diagnosis, complete the IPSS, DIPSS, DIPSS-PLUS, and MIPSS70 scores. Multiple scores indicate that the patient's risk is stratified and assessed as intermediate-risk or higher. According to the guidelines, patients should be treated with rucotinib. The patient is 69 years old, and transplantation is not considered for the time being. Rucotinib is the first choice. A full dose of 20 mg bid is given based on platelet count. As a JAK2 pathway inhibitor, Rucotinib can inhibit cell proliferation on the one hand and relieve physical symptoms on the other. Therefore, taking rocotinib can effectively improve symptoms, shrink the spleen and improve bone marrow pathology. During the treatment, the hemoglobin increased, the platelets remained stable, the spleen shrank significantly, and the MPN-10 symptom score was significantly improved, achieving the goal of treatment and achieving good therapeutic effects.
expert introduction
Deng Xiuzhi
Weihai Municipal Hospital Department of Hematology Chief Physician
[Deng Xiuzhi said the case] An elderly male patient with
, repeated chest tightness and asthma for 1 year, aggravated for a month, physical examination revealed severe anemia, 8 cm below the spleen rib, with pulmonary hypertension, The morphology of bone marrow cells showed teardrop-like red blood cells, extensive proliferation of fibrous tissue on bone marrow biopsy, and grade 2 staining of reticular fibers. Gene test JAK2-V617F was positive. According to the patient's clinical manifestations, physical signs, and auxiliary examinations, it was diagnosed as primary myelofibrosis. PMF is a progressive malignant hematological disease with a median survival time of 5.7 years, and about half of the patients died due to disease progression. If not treated in time, the patient's symptoms can continue to worsen, affecting the quality of life. Treatment is generally based on the prognosis score. If low-risk patients have symptoms, they can be treated with rocotinib; if they have no symptoms, they can be observed. Symptom evaluation is performed every 3 to 6 months. Intermediate-risk -1 patients can be observed, and symptomatic patients can be treated with rucotinib as the first choice. For intermediate-risk -2/high-risk patients, allogeneic hematopoietic stem cell transplantation can be the first choice; if transplantation is not done, if the platelets are more than 50 × 109/L, rucotinib can be given. The prognosis score of this patient is intermediate-risk -2. Rucotinib is the first choice. It has a very good effect. The hemoglobin is increased, the spleen is significantly reduced, and the physical symptoms are significantly improved. Z4z
To sum up, myelofibrosis is a progressive malignant tumor. Malignant clonal gene mutations indicate that patients may be at risk of faster progression. Diagnosis should be accurately stratified. Rucotinib is a targeted inhibitor of JAK pathway, which meets the goals of shrinking the spleen, improving symptoms, prolonging survival and improving bone marrow pathology. In order to maximize the benefits of rucotinib patients, rucotinib can be used as early as the first line, Pay attention to monitor the hematological toxicity during medication, correctly understand the changes, and adjust the dose of rucotinib reasonably. The
MCC number JAK20122298 is valid from December 23, 2021, and the data is expired and deemed invalid.
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content review: Liu Ye, Zhang Jun
title image source: Zhankuhailuo
