{"title":"Safety and efficacy of splenectomy in immune thrombocytopenia.","authors":"Kundan Mishra, Suman Kumar, Rajeev Sandal, Aditya Jandial, Kamal Kant Sahu, Kanwaljeet Singh, Ankur Ahuja, Venkatesan Somasundaram, Rajiv Kumar, Rajan Kapoor, Sanjeevan Sharma, Jasjit Singh, Uday Yanamandra, Satyaranjan Das, Tathagat Chaterjee, Ajay Sharma, Velu Nair","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Immune Thrombocytopenia (ITP) is characterized by low platelet counts. Splenectomy has been in practice for the treatment of ITP since the early 20th century. We aimed to analyze the data of ITP patients from our hospital who underwent splenectomy and further present the long-term outcome and safety profile in these patients.</p><p><strong>Method: </strong>This study was a single-center, registry based study conducted at a tertiary care hospital in Northern India. Patients aged 18 years or more, who underwent splenectomy after at least one line of therapy, were included in the study. The primary outcome was the overall response rate (ORR) at one month after splenectomy. Secondary outcomes were sustained response, relapse-free survival, factors affecting the ORR, and adverse events after splenectomy.</p><p><strong>Results: </strong>Forty-five patients of ITP were included in the study. Thirty-six patients underwent splenectomy in the first half (2001-2010), of the study period. The median age of the patients was 38 (19-56) years. The median duration from diagnosis to splenectomy was 1.76 (0.47-2.58) years. The median number of therapy received before splenectomy was 3 (1-6). The overall response rate (ORR) post-splenectomy at day 30 was 89.2% with 61.8% complete response (CR). The ORR was 88.5% at 1-year, with 48.8% CR. The relapse-free survival (RFS) at 5-years was 57.38% (95% Confidence Interval 40.59-71.02%), There was no effect of duration of disease, age, gender, and prior therapy received, on the ORR at one-month. At one year, the platelet response was significantly better in patients who had a CR at one-month than patients who had a partial response at one month. The relapse-free survival was better in patients who achieved CR after 1-month of splenectomy. During the median follow-up of 5.02 (1 month-20 years) years, there were five cases of overwhelming post-splenectomy infection (OPSI). There was no recorded incidence of perioperative mortality, deep vein thrombosis, or mesenteric thrombosis.</p><p><strong>Discussion: </strong>Despite the variation in outcome from different studies, splenectomy gives the best possible long-term treatment-free remission amongst all the available second-line agents. It is also, one of the most financially affordable therapies. Despite advantages, the number of ITP patients undergoing splenectomy has been on the decline and largely attributable to the newer and more effective second-line therapies. There is no pre-surgery variable predicting the ORR after splenectomy.</p><p><strong>Conclusion: </strong>Splenectomy in ITP offers a long-term sustained response at an economical cost.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 4","pages":"361-372"},"PeriodicalIF":0.0,"publicationDate":"2021-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8446833/pdf/ajbr0011-0361.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39431700","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Esther Natalie Oliva, Sarah M Ronnebaum, Omer Zaidi, Dipen A Patel, Salem Abi Nehme, Clara Chen, Antonio M Almeida
{"title":"A systematic literature review of disease burden and clinical efficacy for patients with relapsed or refractory acute myeloid leukemia.","authors":"Esther Natalie Oliva, Sarah M Ronnebaum, Omer Zaidi, Dipen A Patel, Salem Abi Nehme, Clara Chen, Antonio M Almeida","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) is a rapidly progressive hematological malignancy that is difficult to cure. The prognosis is poor and treatment options are limited in case of relapse. A comprehensive assessment of current disease burden and the clinical efficacy of non-intensive therapies in this population are lacking. We conducted two systematic literature reviews (SLRs). The first SLR (disease burden) included observational studies reporting the incidence and economic and humanistic burden of relapsed/refractory (RR) AML. The second SLR (clinical efficacy) included clinical trials (phase II or later) reporting remission rates (complete remission [CR] or CR with incomplete hematologic recovery [CRi]) and median overall survival (mOS) in patients with RR AML or patients with <i>de novo</i> AML who are ineligible for intensive chemotherapy. For both SLRs, MEDLINE<sup>®</sup>/Embase<sup>®</sup> were searched from January 1, 2008 to January 31, 2020. Clinical trial registries were also searched for the clinical efficacy SLR. After screening, two independent reviewers determined the eligibility for inclusion in the SLRs based on full-text articles. The disease burden SLR identified 130 observational studies. The median cumulative incidence of relapse was 29.4% after stem cell transplant and 46.8% after induction chemotherapy. Total per-patient-per-month costs were $28,148-$29,322; costs and health care resource use were typically higher for RR versus non-RR patients. Patients with RR AML had worse health-related quality of life (HRQoL) scores than patients with <i>de novo</i> AML across multiple instruments, and lower health utility values versus other AML health states (i.e. newly diagnosed, remission, consolidation, and maintenance therapy). The clinical efficacy SLR identified 50 trials (66 total trial arms). CR/CRi rates and mOS have remained relatively stable and low over the last 2 decades. Across all arms, the median rate of CR/CRi was 18.3% and mOS was 6.2 months. In conclusion, a substantial proportion of patients with AML will develop RR AML, which is associated with significant humanistic and economic burden. Existing treatments offer limited efficacy, highlighting the need for more effective non-intensive treatment options.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 4","pages":"325-360"},"PeriodicalIF":0.0,"publicationDate":"2021-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8446831/pdf/ajbr0011-0325.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41099642","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"COVID-19 infection in patients with acute leukemia; Istanbul experience.","authors":"Deram Buyuktas, Kadir Acar, Gulsan Sucak, Tayfur Toptas, Irem Kapucu, Huseyin Bekoz, Simge Erdem, Meliha Nalcaci, Figen Atalay, Meltem Olga Akay, Burhan Ferhanoglu","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>Coronavirus disease 2019 (COVID-19) has led to a global pandemic that has also challenged the management of various other life-threatening conditions, such as malignant disorders. In this study, we present the clinical features and treatment outcomes of twenty-seven COVID-19 positive patients with leukemia across seven different centers in Istanbul. From March 1st to December 31st 2020, 116 patients were diagnosed with acute leukemia. Thirty-two cases with acute lymphocytic leukemia (ALL), 82 cases with acute myeloid leukemia (AML), and 2 cases with mixed phenotype acute leukemia (MPAL) were identified. Of the 27 patients with the COVID-19 infection, seven patients had ALL, 19 patients had AML and one patient had MPAL. The mortality rate was 37% among the patients with AML, whereas there were no deaths in the ALL group. The mortality rate of AML patients with the COVID-19 infection was higher compared to cases without the infection (P<0.05). We could not detect any significant difference in the ALL cohort. This study, which includes one of the largest acute leukemia series in literature proved that acute myeloid leukemia patients with the COVID-19 infection have worse outcomes than patients without the infection. The high mortality among patients with acute leukemias hospitalized with COVID-19 highlight the need for aggressive infection prevention, increased surveillance and protective isolation and even modification of the therapy, in case of minimal residual disease (MRD) negativity.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 4","pages":"427-437"},"PeriodicalIF":0.0,"publicationDate":"2021-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8446830/pdf/ajbr0011-0427.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39430663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Emanuele Cencini, Bianca Mecacci, Francesca Morelli, Francesco Ghio, Ilaria Romano, Silvia Birtolo, Federico Simonetti, Valentina Zoi, Sabrina Moretti, Emanuela Sant'Antonio, Annarosa Cuccaro, Simone Santini, Sofia Kovalchuk, Sara Galimberti, Monica Bocchia, Alberto Fabbri
{"title":"Ibrutinib in patients with relapsed/refractory mantle cell lymphoma: a real-life, retrospective, multicenter trial on behalf of the \"RTL\" (regional Tuscan lymphoma network).","authors":"Emanuele Cencini, Bianca Mecacci, Francesca Morelli, Francesco Ghio, Ilaria Romano, Silvia Birtolo, Federico Simonetti, Valentina Zoi, Sabrina Moretti, Emanuela Sant'Antonio, Annarosa Cuccaro, Simone Santini, Sofia Kovalchuk, Sara Galimberti, Monica Bocchia, Alberto Fabbri","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Relapsed or refractory (R/R) mantle-cell lymphoma (MCL) patients have a poor prognosis and their management is challenging, in absence of a golden standard as salvage treatment. Bruton's tyrosine kinase inhibitor ibrutinib represents an effective treatment for R/R MCL patients. We investigated ibrutinib efficacy and safety in daily clinical practice, together with factors that could predict disease outcome.</p><p><strong>Patients and methods: </strong>We retrospectively analyzed 69 consecutive R/R MCL patients managed in 10 Tuscan onco-hematological centers. The treatment regimen consisted of oral, continuous, single-agent ibrutinib, maximum dosage of 560 mg once per day, until disease progression.</p><p><strong>Results: </strong>Overall response rate was 62.3%, with a CR rate of 39.1%. After a median follow-up of 15.6 months, 40/69 patients (58%) were alive, the main cause of death was progressive disease (PD, 22/69 cases, 31.9%). Median progression-free survival (PFS) and overall survival (OS) were 17 and 34.8 months. Inferior PFS was associated with >1 prior line of therapy and B symptoms. Ibrutinib refractoriness was associated with inferior OS, median OS after ibrutinib failure was only 5 months.</p><p><strong>Discussion and conclusion: </strong>In this real-life setting ibrutinib treatment prolonged survival in R/R MCL patients, without unexpected adverse events. Patients receiving ibrutinib as 2<sup>nd</sup> line regimen had the most favorable outcome.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 4","pages":"373-383"},"PeriodicalIF":0.0,"publicationDate":"2021-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8446834/pdf/ajbr0011-0373.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39431702","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Andrew G Gianoukakis, Shelly Gupta, Theresa N Tran, Patrick Richards, Marelle Yehuda, Sarah E Tomassetti
{"title":"Graves' disease patients with iron deficiency anemia: serologic evidence of co-existent autoimmune gastritis.","authors":"Andrew G Gianoukakis, Shelly Gupta, Theresa N Tran, Patrick Richards, Marelle Yehuda, Sarah E Tomassetti","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>Graves' disease (GD) has been associated with iron deficiency anemia (IDA). Atrophic gastritis leads to IDA and has been associated with autoimmune thyroid disease. This study prospectively determined the prevalence of atrophic gastritis markers and the relationship between these markers and markers of IDA in GD subjects.</p><p><strong>Methods: </strong>Newly diagnosed GD patients (90) and controls (41) were studied. Of the newly diagnosed GD patients, 65 were consecutively enrolled and identified with GD irrespective of anemia, 25 had GD and IDA. Thyroid function, hematologic indices, and atrophic gastritis markers [parietal-cell antibodies (PCab), <i>Helicobacter pylori</i> antibodies (<i>H. pylori</i> ab), mean serum gastrin levels] were examined.</p><p><strong>Results: </strong>GD patients presenting with IDA were twice as likely (64% vs. 32%, P=0.049) to harbor PCabs when compared to all other GD subjects. Unselected GD subjects (n=65) had significantly higher PCab (37% vs. 7%, P<0.001) compared to controls. Gastrin levels were significantly elevated in all GD subjects compared to controls (105 vs. 39 pg/ml, P<0.0001). This difference was magnified in PCab+ subjects (202 vs. 64 pg/ml, P=0.003). In all GD subjects, PCabs were associated with increased gastrin levels (202 vs. 75 pg/ml, P=0.0004) and lower ferritin levels (52 vs. 95, P=0.05). In GD anemic subjects, PCabs were associated with lower mean corpuscular volume (75 vs. 81, P=0.001). Gastrin levels correlated inversely with ferritin levels in all GD subjects and positively with TIBC in GD anemic subjects.</p><p><strong>Conclusions: </strong>A significant subset of patients presenting with GD may suffer from IDA due to concurrent autoimmune atrophic gastritis.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 3","pages":"238-247"},"PeriodicalIF":0.0,"publicationDate":"2021-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8303011/pdf/ajbr0011-0238.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39254654","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ehab Hamed, Mohamed Ahmed Syed, Bayan Faleh Alemrayat, Syed Hammad Anwar Tirmizi, Ahmed Sameer Alnuaimi
{"title":"Haemoglobin cut-off values for the diagnosis of anaemia in preschool-age children.","authors":"Ehab Hamed, Mohamed Ahmed Syed, Bayan Faleh Alemrayat, Syed Hammad Anwar Tirmizi, Ahmed Sameer Alnuaimi","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Background: </strong>The World Health Organisation (WHO) suggests haemoglobin that (Hgb) cut-off levels below 2SD from the population mean to initiate anaemia investigations. In the absence of epidemiological data, Hgb less than 11 g/dL is considered abnormal in children up to the age of 59 months (4 years and eleven months).</p><p><strong>Objectives: </strong>This study reports on the Hgb cut-off levels among children at 1 and 4 years of age. The study compared the prevalence based on the WHO generic cut-off levels and population-specific cut-off-based value defined as below 2SD from the population mean.</p><p><strong>Design, settings, and participants: </strong>A cross-sectional record-based study of healthy children below the age of 59 months attending primary care settings in Qatar. 3 years of Hgb data were collected and analysed using descriptive analyses. We excluded children with any pre-existing disease or who have altered biological parameters indicating a non-healthy child.</p><p><strong>Results: </strong>39407 Participants were stratified into different sub-groups according to age, gender, and ethnicity. Hgb levels were expressed as the mean ± 2SD for children of one and four years of age. Most children were from Western Asia (45.6%), followed by Northern Africa (23.7%), and Southern Asia (21.7%). Our findings for one-year-old children cut-off levels for anaemia might be as low as 9.9 g/dL and 10.6 g/dL for 4-years old.</p><p><strong>Conclusion: </strong>Hgb cut-off values may be set at higher levels for one-year and four-year age groups and many different ethnicities. Higher cut-off points may overestimate the problem as a public health issue. Children may be unnecessarily treated with iron or have needless investigations.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 3","pages":"248-254"},"PeriodicalIF":0.0,"publicationDate":"2021-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8303014/pdf/ajbr0011-0248.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39254655","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Nivolumab in relapsed/refractory Hodgkin lymphoma: towards a new treatment strategy?","authors":"Emanuele Cencini, Monica Bocchia, Alberto Fabbri","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>Chemo-refractory Hodgkin lymphoma (HL), especially after failure of high-dose therapy and autologous stem cell transplantation (ASCT), has a very poor prognosis. Nivolumab, an anti-PD-1 monoclonal antibody, demonstrated durable responses and manageable toxicity in a significant proportion of HL patients who fail both ASCT and brentuximab vedotin. Although anti-PD-1 treatment is often well tolerated, immune-related adverse events (iAE) were frequently observed. New perspectives could be represented by treatment discontinuation in patients with prolonged response or toxicity with the possibility of a re-treatment at relapse, subsequent chemotherapy or a modification of the dose-intensity or treatment duration. The efficacy of anti-PD-1 re-treatment was demonstrated in several cases and we have successfully managed 1 case with this strategy. With the main aim of avoiding the relapse-related psychophysical stress for the patient with manageable toxicity, we have successfully administered nivolumab every 4 weeks to 3 patients in prolonged complete remission, who presented with iAE during treatment. We believe that nivolumab should not only represent a bridge to allogeneic SCT, but it may play an important role also beyond the approved indication and current standard clinical care.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 3","pages":"261-265"},"PeriodicalIF":0.0,"publicationDate":"2021-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8303013/pdf/ajbr0011-0261.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39254657","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"SARS-CoV-2 infection in a pediatric acute leukemia patient on chemotherapy and concurrent sofosbuvir/velpatasvir for HCV.","authors":"Amitabh Singh, Akriti Gera, Aroonima Misra, Sumit Mehndiratta","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>There are new targets identified by experimental and animal research for treatment of SARS-COV-2 (Severe acute respiratory syndrome-Corona Virus-2) infection. Out of many clinical trials registered, there are ongoing human studies highlighting Sofosbuvir's possible role in the treatment of Covid-19 (Coronavirus Disease 2019). Here we present a case of acute leukemia on directly acting antiviral therapy (DAAs) for HCV infection mitigating SARS-COV-2 infection in a patient undergoing chemotherapy. The child was undergoing chemotherapy, along with directly acting antiviral for acute hepatitis C infection. He initially had features of hypoxia and radiological evidence of covid-19. He had an uneventful course and tested negative ten days after onset of illness. With ongoing trials on Sofosbuvir in covid 19 treatment, our finding, albeit coincidental, points to the possible role even in immune-compromised children.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 3","pages":"286-289"},"PeriodicalIF":0.0,"publicationDate":"2021-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8303012/pdf/ajbr0011-0286.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39254621","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Hypomethylating agents+venetoclax induction therapy in acute myeloid leukemia unfit for intensive chemotherapy - novel avenues for lesser venetoclax duration and patients with baseline infections from a developing country.","authors":"Sumeet Mirgh, Archana Sharma, Mohammad Rizwan Mohammad Anwar Shaikh, Kirti Kadian, Narendra Agrawal, Vishvdeep Khushoo, Pallavi Mehta, Rayaz Ahmed, Dinesh Bhurani","doi":"","DOIUrl":"","url":null,"abstract":"<p><p>Both elderly acute myeloid leukemia (AML) patients and those with baseline infections, when treated with intensive chemotherapy, are associated with high induction mortality. We report 24 patients (16-newly-diagnosed, 8-relapsed/refractory) with AML deemed unfit for intensive chemotherapy (by virtue of age >60 years, ECOG-PS 3-4, or those with non-resolving infections at baseline), treated with azacytidine-venetoclax combination as induction chemotherapy. Median follow-up of the study group was 8 months. The overall complete remission (CR)+CR with incomplete count recovery (CRi) rate was 58.3%. 1-year progression-free survival and overall survival of the whole cohort was 44.4% and 55.8%, respectively. On subgroup analysis, newly-diagnosed AML (p=0.05), intermediate-risk cytogenetics (p=0.007), and HMA-naïve (p=0.05) patients had a significantly better outcome. AML patients with baseline infections (versus without infections) treated with azacytidine-venetoclax induction, have lesser induction mortality (compared with historic intensive chemotherapy) with equivalent response rates. A detailed analysis amongst cohorts with different venetoclax durations revealed that, shorter duration (<21 days) venetoclax (versus 21-28 days duration) in induction therapy leads to similar response rates and similar severity of myelosuppression, however, with early count recovery and lesser duration of intravenous antibiotics.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 3","pages":"290-302"},"PeriodicalIF":0.0,"publicationDate":"2021-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8303019/pdf/ajbr0011-0290.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39254622","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Istemi Serin, Mehmet Hilmi Dogu, Gulben Erdem Huq, Osman Yokus
{"title":"A new FLT3 inhibitor with two cases: the gilteritinib experience.","authors":"Istemi Serin, Mehmet Hilmi Dogu, Gulben Erdem Huq, Osman Yokus","doi":"","DOIUrl":"","url":null,"abstract":"<p><strong>Introduction: </strong>In acute myeloid leukemia (AML), a heterogeneous group of leukemias, there are various factors to determine prognosis. Among these prognostic factors, cytogenetic results are increasing in importance day by day. FLT3 mutations are among the most common molecular abnormalities in AML, patients with recurrent or refractory (R/R) AML with this mutation have a low response rate to salvage therapy. Gilteritinib has activity against FLT3, ALK and AXL. This article shall present two cases, for which Gilteritinib was used, a new FLT3 inhibitor, and the results of the treatment. Case 1: A 52-year-old female patient presented to the emergency clinic with weakness and fever. In initial biochemical analysis, leukocyte was 104000/mm<sup>3</sup>. Peripheral smear contained diffuse myeloid blastoid cells, peripheral blood flow cytometry also supported the AML M0-1 phenotype. The bone marrow biopsy aspiration performed on the 14<sup>th</sup> day of induction \"3+7\" treatment, contained diffuse blastic infiltrate and supported refractory disease. In addition to the FLAG-IDA salvage regimen, 120 mg/day Gilteritinib was also started. Bone marrow aspiration performed on the 28<sup>th</sup> day of salvage therapy was compatible with remission. Case 2: 53 years old male patient with also no comorbidity other than known hypertension. In the initial biochemical analysis of the patient, leukocyte was 156000/mm<sup>3</sup>, platelet 58000/mm<sup>3</sup> and hemoglobin 7.6 g/dl. Peripheral blood flow cytometry supported the AML M5 phenotype, whose peripheral smear showed diffuse monoblastoid cells. On the 14<sup>th</sup> day of the patient's 3+7 induction treatment, the control bone marrow aspiration showed diffuse blast infiltration and was considered refractory, FLAG-IDA salvage therapy with again 120 mg/day Gilteritinib per oral were started. On the 28<sup>th</sup> day, control bone marrow aspiration was evaluated as remission.</p><p><strong>Discussion and conclusion: </strong>Unlike other FLT 3 inhibitors, Gilteritinib has been shown to be a highly effective agent in R/R AML with FLT3 mutations. Being the first data to be reported from Turkey, we think it would be quite guiding the titular.</p>","PeriodicalId":7479,"journal":{"name":"American journal of blood research","volume":"11 3","pages":"271-278"},"PeriodicalIF":0.0,"publicationDate":"2021-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8303015/pdf/ajbr0011-0271.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39254659","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}