Leukemia Research Reports最新文献

{"title":"Successful treatment of AML using non-intensive chemotherapy in Jehovah's Witness patients","authors":"David Page,&nbsp;Daniel Sawler,&nbsp;Joseph Brandwein","doi":"10.1016/j.lrr.2024.100477","DOIUrl":"10.1016/j.lrr.2024.100477","url":null,"abstract":"<div><p>Acute myeloid leukemia (AML) patients undergoing induction chemotherapy receive transfusion support to manage severe cytopenias and associated sequelae. Jehovah's Witness (JW) patients typically decline transfusion of most or all blood products. This can lead to exclusion of JW patients from otherwise life-saving treatments due to safety concerns. We present two cases demonstrating the successful induction of JW patients without the need for red cell or platelet transfusion support; the first, an older AML patient induced with azacitidine &amp; venetoclax; the second, a patient with acute promyelocytic leukemia induced using arsenic trioxide and all-trans retinoic acid. Both patients required modifications to the induction regimens to accommodate their wishes. These cases support growing evidence that selected JW patients with AML can be successfully treated using appropriate accommodations.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100477"},"PeriodicalIF":0.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000670/pdfft?md5=a4e1f10b090b2b1a38c29e7419937478&pid=1-s2.0-S2213048924000670-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141985311","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":"First-line therapy with daratumumab, lenalidomide and dexamethasone for patient with POEMS syndrome: A case report","authors":"E. Amabile,&nbsp;F. Fazio,&nbsp;M. Martelli,&nbsp;MT. Petrucci","doi":"10.1016/j.lrr.2024.100491","DOIUrl":"10.1016/j.lrr.2024.100491","url":null,"abstract":"<div><div>POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, M–protein, skin changes syndrome) is a rare condition due to an underlying plasma cell neoplasm whose clinical presentation can be various so it could lead to delayed diagnosis and treatment. The pathogenesis of the syndrome is not well understood, and its therapy is adapted from other plasma cell disorders with the aim of alleviating symptoms, decreasing end-organ damage, improving quality of life and prolonging overall survival. We report a case of a 71 years-old woman who has been treated with continuous DRd (daratumumab, lenalidomide and dexamethasone) scheme.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100491"},"PeriodicalIF":0.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142744788","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":"PATHOGENIC MECHANISMS OF DDX41 MUTATIONS IN THE DEVELOPMENT OF MYELOID MALIGNANCIES","authors":"A. Kon ,&nbsp;M. Nakagawa ,&nbsp;A. Tomita ,&nbsp;K. Kataoka ,&nbsp;N. Kakiuchi ,&nbsp;H. Makishima ,&nbsp;M. Nakayama ,&nbsp;H. Koseki ,&nbsp;Y. Nannya ,&nbsp;S. Ogawa","doi":"10.1016/j.lrr.2024.100425","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100425","url":null,"abstract":"<div><h3>Introduction</h3><p>Germline <em>DDX41</em> variants are implicated in late-onset myeloid neoplasms, accounting for the largest germline risk of the development of myeloid neoplasms. In typical cases, a germline loss-of-function allele is compounded by the somatic R525H mutation affecting the helicase domain in the remaining allele. The molecular mechanism by which <em>DDX41</em> mutations lead to myeloid neoplasms remains to be elucidated.</p></div><div><h3>Methods</h3><p>To delineate the pathogenic mechanism of <em>DDX41</em>-mutated myeloid neoplasms, we generated mice models carrying the conditional <em>Ddx41</em> knock-out and/or R525H knock-in alleles.</p></div><div><h3>Results</h3><p>In non-competitive bone marrow (BM) transplantation, most of the mice reconstituted with <em>Ddx41</em>^−/− or <em>Ddx41</em>^R525H/− BM died within a month due to severe BM failure. In competitive transplantation, <em>Ddx41</em>^−/− and <em>Ddx41</em>^R525H/− mice-derived cells showed markedly disadvantageous reconstitution, while <em>Ddx41</em>^+/− and <em>Ddx41</em>^R525H/+ mice-derived cells showed slightly reduced reconstitution compared to <em>Ddx41</em>^+/+ mice-derived cells. By contrast, the mice transplanted with <em>Ddx41</em>^+/− or <em>Ddx41</em>^R525H/+ BM showed significantly reduced WBC counts and anemia in long-term observation in both primary and serial transplantations. Some of the <em>Ddx41</em>^+/− or <em>Ddx41</em>^R525H/+ BM-transplanted mice exhibited MDS-like phenotypes, showing ineffective hematopoiesis with evidence of erythroid dysplasia. Transcriptome analysis of <em>Ddx41</em>^+/− and <em>Ddx41</em>^R525H/+ derived stem cells exhibited a significant deregulation of genes involved in RNA metabolism, ribosome biogenesis and apoptosis.</p></div><div><h3>Conclusions</h3><p>Monoallelic <em>Ddx41</em> loss-of function or R525H knock-in alleles led to age-dependent impaired hematopoiesis and the development of myeloid malignancies, while compound biallelic loss-of function and R525 alleles showed a severely compromised function of hematopoietic stem cells.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100425"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000153/pdfft?md5=fedf7d34fa8db23043870c6d996d5179&pid=1-s2.0-S2213048924000153-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103290","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":"CYTOGENOMIC PROFILING OF LARGE COHORT OF INDIAN MYELODYSPLASTIC SYNDROMES","authors":"N. Maurya ,&nbsp;C. Shanmukhaiah ,&nbsp;M. Madkaikar ,&nbsp;B.R. Vundinti","doi":"10.1016/j.lrr.2024.100427","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100427","url":null,"abstract":"<div><h3>Introduction</h3><p>About 40% - 50% of Myelodysplastic syndromes (MDS) patients present with a normal karyotype whereas 50% - 60% of patients are of low risk, however, they tend to have worse outcomes and a higher chance of disease progression. Hence, we aim to study the genomic pathophysiology of disease and their association with overall survival in MDS.</p></div><div><h3>Methods</h3><p>The study cohort included 200 MDS patients. The molecular cytogenetic and mutation profiling was carried out in all MDS patients. The patients were clinically followed up. The Kaplan-Meier survival and multivariate analysis was performed using GraphPad Prism 5.00</p></div><div><h3>Results</h3><p>The WHO 2016 classification revealed a high frequency of MDS – MLD (33%) followed by MDS – SLD (25%), MDS – EB-1/2 (24%). The chromosomal aberrations were identified in 35% of MDS patients by CK/FISH. The NGS identified gene mutations in 75% of the cohort. The survival analysis revealed that the mutations in <em>TP53, JAK2/3, KRAS, NRAS</em>, and <em>ASXL1</em> are significantly (<em>P</em> &lt; 0.05) associated with poor survival. SNP array analysis (n=77), revealed, patients with chromosome 2 aberrations have a poor prognosis. SNP array combined with NGS confirmed the biallelic loss of function of the TP53 gene (3/7), a clinically relevant biomarker and new genetic-based MDS entity i.e. MDS-biTP53 as per the new WHO classification 2022. The univariate and multivariate analysis suggested that genetic lesions (mutations/CNVs/LOH) with IPSS-R could be prognosticating markers in MDS patients.</p></div><div><h3>Conclusions</h3><p>Our study strongly supports that the genome profiling by combined CGH+SNP array and NGS improves prognostication and hence personalized disease management.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100427"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000177/pdfft?md5=0a96e00835b0036ad839333d54d0b25f&pid=1-s2.0-S2213048924000177-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103292","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":"THE ROLE AND MECHANISM OF UPREGULATED TYPE I INTERFERON SIGNALING PATHWAY IN THE PATHOGENESIS OF CHRONIC MYELOMONOCYTIC LEUKEMIA","authors":"L. Na,&nbsp;S. Yuqian","doi":"10.1016/j.lrr.2024.100429","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100429","url":null,"abstract":"<div><h3>Introduction</h3><p>Chronic Myelomonocytic Leukemia (CMML) is a rare, malignant hematopoietic system tumour whose pathogenesis remains unclear. This primarily affects elderly males and may transform into acute myeloid leukemia. Elevated innate immune and inflammation-related pathways were found in CMML patients' monocytes. Activation of type I interferon signalling pathway was significant and correlated with prognosis. Strong expression of interferon-regulated genes in patients pointed to type 1 interferon signalling's role in CMML's pathogenesis. TET2 and SRSF2 mutations, affecting gene expression and cellular function, are common in CMML, with a synergistic effect shown in mouse model experiments.</p></div><div><h3>Methods</h3><p>We studied 14 untreated CMML patients, analysing their peripheral blood monocytes and 13 inflammatory cytokines via transcriptome sequencing and Cytometric Bead Array. SRSF2-P95H/TET2 mutation cell lines were also created.</p></div><div><h3>Results</h3><p>Exome and transcriptome sequencing in 14 CMML patients revealed frequent TET2, ASXL1, and SRSF2 mutations. Compared to controls, CMML cells displayed activated innate immune and inflammation pathways, including elevated levels of IL-10, IFN-α2, IL-8, IL-12p70, IL-6, and IL-17A. Higher 1-IFN scores, indicating the activation level of the type 1 interferon pathway, were seen in MP-CMML patients, suggesting a link with poor prognosis. In vitro, interferon pathway inhibition induced apoptosis in CMML monocytes and reduced protein P38 phosphorylation, inhibiting cell proliferation in THP-1/U937. TET2 loss-of-function mutations and SRSF2-P95H mutations overexpressed type 1 interferon pathway genes, leading to increased culture supernatant interferon levels in HEK-293T cells.</p></div><div><h3>Conclusions</h3><p>Study shows TET2 loss-of-function/SRSF2-P95H mutations in CMML trigger type I interferon pathway activation, promoting P38 and PI3K phosphorylation, potentially partly causing CMML.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100429"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000190/pdfft?md5=07280409c9a88f59574c7669641476da&pid=1-s2.0-S2213048924000190-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103589","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":"GENETIC LANDSCAPE OF MYELOID NEOPLASMS IN PATIENTS WITH FANCONI ANEMIA","authors":"K. Yoshida ,&nbsp;M. Yabe ,&nbsp;N. Kakiuchi ,&nbsp;M. Takata ,&nbsp;K. Katayama ,&nbsp;S. Imoto ,&nbsp;S. Ogawa ,&nbsp;H. Yabe","doi":"10.1016/j.lrr.2024.100439","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100439","url":null,"abstract":"<div><h3>Introduction</h3><p>Fanconi anemia (FA) is an inherited bone marrow failure syndrome characterized with chromosomal instability and a high propensity to myeloid malignancy. Although causative genes have been identified in most patients, the genetic background of the leukemic transformation in FA has not been fully understood.</p></div><div><h3>Methods</h3><p>We studied 2 acute myeloid leukemia (AML) and 7 myelodysplastic syndromes (MDS) developed in patients with FA using whole-genome sequencing and analyzed somatic mutations, structural variants and copy number alterations.</p></div><div><h3>Results</h3><p>The number of somatic mutations and copy number alterations (CNAs) in AML were 1,071 mutations and 4 CNAs per patient on average, which tended to be higher than those in MDS (265 mutations and 2.3 CNAs per patient). For mutational signatures, three known signatures were identified, which included SBS1 and SBS5 caused by endogenous mutational processes, and SBS3 related to defective homologous recombination. Mutations and structural variants affected known driver genes in myeloid malignancies, such as <em>RUNX1</em> (n = 3), <em>ASXL1</em> (n = 1), <em>CBL</em> (n = 1), <em>NRAS</em> (n = 1) and <em>KDM6A</em> (n = 1). Recurrent copy number alterations were more frequently detected, including +3q (n = 6), +1q (n = 4) and -7q (n = 2). The majority of these CNAs were clonal and all but one patient harbored either of +3q or +1q, indicating the early acquisition of copy number changes and their driver role in leukemic transformation.</p></div><div><h3>Conclusions</h3><p>Myeloid neoplasms related with FA were characterized by a unique pattern of CNAs and common driver mutations in myeloid malignancies.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100439"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000293/pdfft?md5=431471d8f80b44a6224ac1d184cf4c09&pid=1-s2.0-S2213048924000293-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103875","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":"Successful treatment of a CLL associated IgM hyper-viscosity syndrome: A rare case","authors":"Toufic Tannous ,&nbsp;Gil Hevroni ,&nbsp;Raiyan Islam ,&nbsp;Georgios Pongas","doi":"10.1016/j.lrr.2024.100479","DOIUrl":"10.1016/j.lrr.2024.100479","url":null,"abstract":"<div><p>In the context of chronic lymphocytic leukemia (CLL), Hyperviscosity Syndrome (HVS) typically arises from hyperleukocytosis, although it infrequently stems from IgM hyperparaproteinemia. We present a distinctive case of HVS induced by IgM hyperparaproteinemia in a patient experiencing relapsed CLL, marked by bulky disease and cytopenias upon progression. The patient exhibited new symptoms, including headache, dizziness, and confusion. Laboratory analysis revealed an elevated total protein level, and serum electrophoresis identified an elevated M spike at 4 g/dL with IgM on immunofixation. Suspecting HVS, prompt plasmapheresis was initiated, resulting in symptom resolution within two days.</p><p>A comprehensive literature review suggests that CLL patients with an elevated IgM level often face a poor prognosis, though HVS symptoms are not commonly observed. Our case underscores the significance of swiftly identifying HVS when IgM hyperparaproteinemia is detected in CLL patients. Notably, our patient not only achieved successful treatment for the acute presentation but also initiated second-line therapy for relapsed disease. In conclusion, effective management and stabilization of CLL patients with IgM-associated HVS are attainable, emphasizing the crucial role of prompt recognition.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100479"},"PeriodicalIF":0.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000694/pdfft?md5=5e85418b438fce9432b4f1809324a226&pid=1-s2.0-S2213048924000694-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142076957","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":"A 95-year-old patient suffered high-grade B-cell lymphoma combined hairy cell leukemia","authors":"Lingli Wang ,&nbsp;Jing Wang ,&nbsp;Min Li ,&nbsp;Lei Tian","doi":"10.1016/j.lrr.2024.100457","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100457","url":null,"abstract":"<div><p>This case report discusses a 95-year-old man diagnosed with two types of lymphomas. He was hospitalized for erysipelas in September 2018. The lymph node revealed high-grade B-cell lymphoma with Myc and Bcl-2 rearrangement. Bone marrow biopsy revealed hairy cell leukemia, a rare type of indolent B-cell lymphoma. We found that the bone marrow and left inguinal lymph node were non-homologous. There are no known reports of super-aged patients with two types of lymphoma simultaneously. The toxicity of R-CHOP in elderly people limited its usage, so we first chose rituximab. However, this approach was not successful. We then considered the Bruton tyrosine kinase (BTK) inhibitor, but its use was limited due to high blood pressure. Finally, we administered venetoclax, which the patient took for 2 years. The results of the routine blood examination were close to normal and no enlarged superficial or abdominal lymph nodes were observed.This is the oldest reported patient with two types of malignant lymphatic diseases. Additionally, this rare case suggests that targeted therapy can be more effective and safe for super-aged individuals. To summarize, a 95-year-old man diagnosed with two types of lymphomas, high-grade B-cell lymphoma and hairy cell leukemia, was successfully treated with venetoclax after other treatments failed. This case suggests that targeted therapy can be effective and safe for super-aged patients with multiple malignant lymphatic system diseases.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100457"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000475/pdfft?md5=c400f23e6944029eca93b5743974a2be&pid=1-s2.0-S2213048924000475-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140295824","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":"Extramedullary plasmacytoma of the orbit complicating the evolution of multiple myeloma in complete remission","authors":"Nader Slama ,&nbsp;Inaam Bizid ,&nbsp;Ahlem Bellalah ,&nbsp;Mabrouk Abdelali ,&nbsp;Mohamed Adnene Laatiri","doi":"10.1016/j.lrr.2024.100460","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100460","url":null,"abstract":"<div><p>Orbital plasmacytoma is rare and has only been reported in the context of the initial diagnosis of multiple myeloma. Moreover, isolated orbital plasmacytoma without any signs of multiple myeloma is extremely rare.</p><p>We report the case of a 59-year-old female patient diagnosed with IgA Kappa multiple myeloma. It was stage I ISS (International Staging System) and stage I R-ISS (Revised ISS). According to the Tunisian national protocol, the patient was included in the standard-risk group and was eligible for four cycles of CTD (Cyclophosphamide, Thalidomide, Dexamethasone) followed by autologous stem cell transplantation. Taking into account the partial response after the CTD cycles, the patient has benefited from two VTD cycles (Bortezomib, Thalidomide, Dexamethasone). Thus, complete remission was obtained. The patient refused autologous stem cell transplantation. Therefore, maintenance treatment based on Thalidomide only was started and received over a twelve-month period.</p><p>Five months after the end of maintenance treatment, she reported frontal headaches that were resistant to symptomatic treatment, with ptosis in the right eye in physical examination. Brain MRI revealed the presence of a right cranio-orbital tissue mass with intra-orbital and extra-axial cerebral components. The mass measured 32/36 mm on axial sections and 47 mm in height. The patient underwent a complete resection of the cranio-orbital mass with cranioplasty. The histopathological examination of the mass with Immunohistochemistry staining confirmed the diagnosis of orbital plasmocytoma.</p><p>An update of the multiple myeloma assessment did not reveal any biological, cytological or radiological signs in favor of multiple myeloma. Therefore the diagnosis of isolated orbital plasmacytoma without signs of multiple myeloma was made.</p><p>Post-operative brain MRI showed complete disappearance of the right cranio-orbital tissue lesion. There was only a persistent meningeal enhancement of the dura mater at the surgical site, suggestive of post-operative changes. The patient was then referred for cranio-orbital radiotherapy.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100460"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000505/pdfft?md5=6b41ef9dd4242995d540915531566917&pid=1-s2.0-S2213048924000505-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140646740","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":"Challenges and practical considerations in the management of blastic plasmacytoid dendritic cell neoplasm: A single-center experience","authors":"Haripriya Andanamala ,&nbsp;Naveen Pemmaraju ,&nbsp;Taha Al-Juhaishi","doi":"10.1016/j.lrr.2024.100486","DOIUrl":"10.1016/j.lrr.2024.100486","url":null,"abstract":"<div><div>Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is a rare and often life-threatening complex hematologic malignancy that can commonly infiltrate the skin, lymph nodes, central nervous system, and bone marrow. In the setting of the infrequency of confirmed diagnoses of BPDCN, and given limited prospective studies, it has been associated with lackluster outcomes with a median overall survival between 9 and 23 months. We herein discuss our experience treating five consecutive patients with BPDCN at our center since the approval of TAG. We also present some of the challenges that face oncology providers treating this disease due to exceptional rarity and aggressive nature of this disease in addition to overall limited experience and effective therapies</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100486"},"PeriodicalIF":0.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142660222","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}