A case report of ruxolitinib reversing myelofibrosis

Abstract

Abstract This abstract reports the diagnosis and treatment process of a patient with primary myelofibrosis (PMF). Patient, Huang xx, female, born in October 1976, presented with fatigue and abdominal distension in October 2003 and was diagnosed with Primary Myelofibrosis（PMF）. Bone marrow pathology examinations both in 2007 and 2014 showed significant proliferation of fibrous tissue (++). During this period, JAK2 V617F was tested positive in May 2010. The patient received sequential treatments with hydroxyurea, interferon, etc., but there was no retraction of the spleen, and symptoms such as fatigue and abdominal distension gradually worsened, seriously affecting the quality of life and labor ability. In May 2017, the patient developed left upper abdominal pain, night sweats, and weight loss. The spleen extended 16 cm below the left costal margin into the pelvic cavity. Routine blood tests showed: WBC 6.5×10⁹/L, HGB 113 g/L, PLT 494×10⁹/L. Bone marrow pathology examination indicated MF-3. Treatment with ruxolitinib 15 mg twice daily was initiated on July 28, 2017. Three months later, symptoms such as abdominal pain and night sweats improved significantly. The MPN-10 score decreased from 46 points before treatment to 36 points, and the spleen retracted to 5 cm below the costal margin. After 15 months of continuous ruxolitinib treatment, the MPN-10 score dropped to 17 points, and the degree of myelofibrosis reduced from MF-3 to MF-1, achieving reversal of myelofibrosis. In November 2024, a re-examination showed that the degree of myelofibrosis remained MF-1, the MPN-10 score decreased to 6 points, and the spleen was stable at 5 cm below the costal margin. During the treatment period, iron deficiency anemia occurred due to menorrhagia, with Hgb dropping to 86 g/L. After iron supplementation therapy, Hgb rose back to 112 g/L. The patient has been receiving ruxolitinib treatment for 8 years, with good general condition, relatively stable blood routine indicators, and the spleen stably maintained at 5 cm below the left costal margin. The main treatment goals of improving symptoms, reducing spleen size, reversing myelofibrosis, and prolonging overall survival have been fully achieved. More fortunately, the patient has received long-term assistance from the ruxolitinib charity program and has been using the medication for free.