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Case 37
Published in Atul B. Mehta, Keith Gomez, Clinical Haematology, 2017
Chronic myelomonocytic leukemia is a slowly progressive condition which is usually treated with oral chemotherapy (e.g. hydroxyurea or etoposide). Splenectomy is helpful if patients become transfusion-dependent or if there are symptoms due to an enlarged organ. Transformation to acute leukemia occurs with a median interval of 18–24 months, but intensive chemotherapy is rarely successful. Younger patients should be considered for intensive therapy, possibly followed by bone marrow transplantation; however, this particular patient did not have a suitable donor 5 azacitidine is often helpful. Recent publications have indicated a role for eltrombopag as an orally active agent.
Principal Side-Effects of the Treatment of Leukemias, Lymphomas, and Myelomas
Published in Tariq I Mughal, John M Goldman, Sabena T Mughal, Understanding Leukemias, Lymphomas, and Myelomas, 2017
Tariq I Mughal, John M Goldman, Sabena T Mughal
The first such drug to receive Food and Drug Administration (FDA) approval in November 1997 was interleukin-11 or oprelvekin (Neumega). It was approved for adult patients with non-myeloid cancers for chemotherapy-related thrombocytopenia based on studies confirming the drug’s efficacy in patients who had experienced severe thrombocytopenia following chemotherapy. In general the drug is indeed quite safe but in early 2009 there has been some concern about changes in visual acuity and visual field defects, possibly related to optic neuropathy, in addition to ventricular arrhythmias. Further tests are now in progress. The other two platelet-stimulating drugs are the subcutaneously administered romiplostim (AMG-531) and the orally administered eltrombopag (Promacta). Both drugs received regulatory approval by the FDA in 2008 for use in chronic immune thrombocytopenia and chemotherapy-induced thrombocytopenia.
Principal Treatment-Related Side Effects
Published in Tariq I. Mughal, Precision Haematological Cancer Medicine, 2018
Myelosuppression is probably the most frequently observed haematological toxicity of cancer therapies in general. Cytotoxic, chemotherapy-associated myelosuppression can often be more serious than that caused by targeted therapies, since febrile neutropenia requiring urgent intervention is not an uncommon event. For most of the drugs, the nadir is at 7 to 10 days, when patients will be at risk of acquiring a bacterial or fungal infection as a result of neutropenia. Early administration of broad-spectrum antibiotics, and in some cases use of G-CSF to promote recovery of the neutrophils, may be indicated. Chemotherapy-associated anaemia is common and usually mild and may not require red cell transfusions. Thrombocytopenia can also occur and, if severe, may require the administration of platelet transfusions, and occasionally a TPO receptor agonists, such as romiplostin or eltrombopag. TKIs, in particular dasatinib, typically lead to a dose-dependent transient neutropenia, but can also result in anaemia and thrombocytopenia. Other manifestations of haematological toxicities include thrombotic-thrombocytopenic purpura/haemolytic-uraemic syndrome with VEGFR inhibitors, such as sunitinib, and bone/bone marrow necrosis, which can be seen in patients receiving steroids, particularly in high doses for long periods, as is often the case in particular in patients with acute lymphoblastic leukaemia. This is irreversible and requires most patients to discontinue the treatment; surgical correction may be indicated. There has been much concern about osteonecrosis of the jaw (ONJ), particularly in patients receiving bisphosphonates (Figure 13.5). B-cell aplasia has also been reported in rare patients receiving immunotherapies.
The efficacy and safety of eltrombopag in treating TKI-induced thrombocytopenia in patients with chronic myeloid leukemia
Published in Hematology, 2023
Li Liu, Yilin Chen, Yan Liang, Li Meng, Jingming Guo, Chuancai Liu, Zhe Zhao, Jing Zou, Wenjuan He, Jiangzhao Zhang, Zhenya Hong, Caixia Liang, Xianjie Fu, Hui Wu, Youshan Zhang, Yanli Zhang, Weiming Li
Domestic and international guidelines recommend granulocyte colony-stimulating factor(G-CSF) as a clinical treatment for TKI-induced neutropenia. In addition, human erythropoietin injection is used clinically for TKI-induced anemia. However, there are fewer therapeutic drugs for TKI-induced thrombocytopenia. Eltrombopag is an oral, synthetic, non-peptide, small-molecule human thrombopoietin agonist used for the treatment of immune thrombocytopenia, aplastic anemia, and hepatitis C-associated thrombocytopenia [8–10]. Recently, a non-rando-mized, phase II, single-arm study in America has established that eltrombopag can relieve TKI-induced thrombocytopenia in CML patients, thereby improving the efficacy of TKI [11]. However, there is a lack of studies in China and the Asia-Pacific region. Therefore, we retrospectively analyzed the clinical data of 21 CML patients treated with eltrombopag for TKI-induced thrombocytopenia to provide a reference for its efficacy and safety in CML patients.
Real-world experience of treatment with thrombopoietin receptor agonists in anti-thymocyte globulin-naïve patients with aplastic anemia: an observational retrospective analysis in a single institution
Published in Hematology, 2022
Masaki Iino, Atsushi Jinguji, Tomoya Sato, Ayato Nakadate
Eltrombopag was initially administered, in accordance with the approved indications and guidelines for patients with AA in Japan based on no increase in reticulocyte count (<20×109/L from baseline) within two months after initiation of CsA monotherapy [9,12,14,15]. If eltrombopag was ineffective or resulted in serious AEs, it was changed to romiplostim at the treating physicians’ discretion. Briefly, eltrombopag was orally administered starting at 25 mg/day; after two weeks of administration, dose adjustments in increments of 25 mg/day were considered, up to a maximum dose of 100 mg/day. Romiplostim was subcutaneously injected starting at 10 μg/kg once a week; this was adjusted in increments of 5 μg/kg up to a maximum of 20 μg/kg, according to hematopoietic recovery [16]. After achievement of stable trilineage responses, the TPO-RA dosage was decreased gradually and then discontinued while trilineage hematopoiesis was sustained. TPO-RA treatment was resumed when loss of response was observed, again at the treating physicians’ discretion. Cytogenetic analysis (G-band staining and fluorescence in situ hybridization [FISH] for chromosome 7q deletion) was performed every 6–12 months or when loss of hematologic response was observed, using bone marrow or peripheral blood samples.
Has Eltrombopag eliminated the need to use allogeneic HSCT in first line treatment of pediatric aplastic anemia?
Published in Pediatric Hematology and Oncology, 2021
Chia Huan Ng, Fraser Jang-Milligan, Kirk R. Schultz
The two recent papers in this issue of Pediatric Hematology Oncology present an improvement in IST outcomes potentially challenging the current recommended treatment algorithm for pediatric AA.4,5 The two publications, both by Chinese groups, showed promising outcomes. Fang et al. reported a cohort of 57 pediatric patients with AA treated with porcine ATG and Eltrombopag; median age of the cohort is 6.5 years. Response rates were significantly higher (Complete response (CR): 50% vs. 17.9%; p < 0.05, Overall response rate (ORR): 94.4% vs. 69.2%, p < 0.05) at 6 months in patients treated with IST and Eltrombopag compared to IST alone. Importantly, the use of Eltrombopag has not led to an increase in clonal evolution resulting in the development of myelodysplasia or hematological malignancies; this further confirms the safety of utilizing Eltrombopag in patients with AA. Jie et al. reported their cohort of 14 pediatric patients with AA treated with rabbit ATG and Eltrombopag. CR and ORR were 64.3% and 78.6%, respectively at 6 months. There were no relapses in the responders. The overall survival for patients who responded to a combination of ATG and Eltrombopag in both of these studies were 100%. Furthermore, Eltrombopag was well tolerated with the most common side effect being liver dysfunction.