Acta Haematologica最新文献

{"title":"Low Rate of Infectious Enterocolitis in Allogeneic Stem Cell Transplant Recipients with Acute Diarrhea: A Prospective Study by the GETH-TC.","authors":"Sara Redondo Velao,&nbsp;Irene Garcia Cadenas,&nbsp;Mª Angeles Cuesta,&nbsp;Isabel Sanchez-Ortega,&nbsp;Francesc Fernández-Avilés,&nbsp;Elisa Roldan,&nbsp;Anna Torrent,&nbsp;M Cruz Viguria,&nbsp;Sara Villar,&nbsp;Leyre Bento,&nbsp;Lucrecia Yañez,&nbsp;Rodrigo Martino,&nbsp;Jose Luis Piñana","doi":"10.1159/000528242","DOIUrl":"https://doi.org/10.1159/000528242","url":null,"abstract":"<p><p>Acute diarrhea is a common and debilitating complication in recipients of allogeneic hematopoietic stem cell transplantation (HCT). In this prospective, observational, and multicenter study we examined all episodes occurring in the first 6 months of 142 consecutive adult patients who underwent a reduced-intensity conditioning HCT in 10 Spanish tertiary university hospitals. Fifty-four patients (38%) developed a total of 75 acute diarrhea episodes. The median time from HCT to the first episode was 38 days (4-157). The main cause of enterocolitis was lower GI-aGVHD (38%), followed by infections (21%) and drug-related toxicity (8%). Causative infectious causes were identified in only 16/75 episodes (21%). C. difficile-related infection was the most common infectious agent with an incidence and recurrence of 13% and 2%, respectively. With a median follow-up for survivors of 32 months, the non-relapse mortality (NRM) and the overall survival (OS) at 1 year, were 20% (95% C.I.: 14-28%) and 69% (95% C.I.: 61-77%), respectively. Development of enterocolitis was not associated with higher NRM (p = 0.37) or worse OS (p = 0.9). This real-life study confirms that the diagnosis and management of acute diarrhea in the early stages after HCT is challenging. Nosocomial infections seem to be relatively uncommon, probably due to more rational use of antibiotics.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 2","pages":"161-165"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10249261","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Erratum.","authors":"","doi":"10.1159/000529163","DOIUrl":"https://doi.org/10.1159/000529163","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 2","pages":"172"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10253124","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study.","authors":"Moshe Mittelman,&nbsp;Uwe Platzbecker,&nbsp;Sebastian Grosicki,&nbsp;Tomasz Lawniczek,&nbsp;Zewen Zhu,&nbsp;Dominik Selleslag","doi":"10.1159/000531146","DOIUrl":"10.1159/000531146","url":null,"abstract":"<p><p>ASPIRE, a three-part, international, phase 2 trial (ClinicalTrials.gov identifier: NCT01440374), investigated eltrombopag efficacy and safety in patients with advanced myelodysplastic syndrome or acute myeloid leukemia and grade 4 thrombocytopenia (&lt;25 × 109 platelets/L). Approximately 30-65% of patients in this open-label extension phase experienced clinically relevant thrombocytopenic events; no conclusions could be made regarding long-term efficacy (non-randomized design, no placebo control), and survival rates may simply reflect advanced disease. Long-term safety was consistent with the double-blind phase and contrasted with earlier SUPPORT study findings in higher-risk patients, suggesting that eltrombopag may have a role in treating thrombocytopenia in patients with low-/intermediate-risk myelodysplastic syndrome.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"373-378"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10614231/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9527227","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"On Behalf of the SFGM-TC: Prophylactic Donor Lymphocyte Infusion in Patients Treated with Allogeneic Stem-Cell Transplantation for High-Risk Myelodysplastic Syndrome and Acute Myeloid Leukemia.","authors":"Charles Guisnel,&nbsp;Luciane Schirmer,&nbsp;Stéphane Morisset,&nbsp;Marie Robin,&nbsp;Hélène Labussière-Wallet,&nbsp;Rémy Duléry,&nbsp;Patrice Ceballos,&nbsp;Edouard Forcade,&nbsp;Stéphanie Nguyen,&nbsp;Xavier Poiré,&nbsp;Johan Maertens,&nbsp;Sylvain Chantepie,&nbsp;Patrice Chevallier,&nbsp;Etienne Daguindau,&nbsp;Alban Villate,&nbsp;Amandine Charbonnier,&nbsp;Cristina Castilla-Llorente,&nbsp;Nathalie Contentin,&nbsp;Anne Huynh,&nbsp;Ibrahim Yakoub-Agha,&nbsp;Claude Eric Bulabois,&nbsp;Marie-Thérèse Rubio,&nbsp;Maud D'Aveni","doi":"10.1159/000528184","DOIUrl":"https://doi.org/10.1159/000528184","url":null,"abstract":"<p><strong>Introduction: </strong>Allogeneic hematopoietic stem-cell transplantation (allo-HSCT) remains the best curative option for high-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Unfortunately, it is still associated with a significant risk of relapse due to mechanisms of escape from the control of alloreactive T cells. Repetitive adjuvant donor lymphocyte infusion (DLI), termed prophylactic DLI (proDLI), as an effective strategy in preventing relapse is still debated.</p><p><strong>Methods: </strong>We performed a retrospective multicenter study to evaluate the efficacy of proDLI in allografted AML and MDS. We identified 56 patients treated with proDLI (DLI planned in full chimeras without any sign of disease relapse) and matched them to 167 patients in control group, (DLI performed for mixed chimerism or positive minimal residual disease) based on similar age, initial disease, cytogenetic prognosis, and conditioning intensity.</p><p><strong>Results: </strong>In univariate analysis, the incidence of severe aGVHD at 100 days and incidence of all grades of chronic GVHD 1 year after allo-HSCT were similar in the two groups. We also observed a trend of higher 3-year RI (52.61% [95% confidence interval 25.99-79.23]) in the proDLI group versus the control group (29.31% [20.28-38.34], p = 0.067). However, 3-year overall survival (p = 0.892), progression-free survival (p = 0.239), and nonrelapse mortality (p = 0.343) were similar between the two groups. In multivariate analysis, the only factor influencing overall and progression-free survival was anti-thymocyte globulin administration during the conditioning regimen.</p><p><strong>Conclusion: </strong>The proDLI strategy had an acceptable toxicity profile but did not improve patient outcomes compared to the pre-emptive strategy.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 3","pages":"230-239"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9925820","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Case Report of Chronic Myelogenous Leukemia Presenting as Blastic Crisis with a T-Cell Acute Lymphoblastic Leukemia Phenotype: Awareness of a Rare Entity.","authors":"Maria Efstathopoulou, Katerina Zoi, Marina P Siakantaris, Daphne Koumbi, Anna Zannou, Evangelia-Faidra Triantafyllou, Gerassimos Tsourouflis, Eleftheria Lakiotaki, Theodoros P Vassilakopoulos, Maria K Angelopoulou","doi":"10.1159/000529911","DOIUrl":"10.1159/000529911","url":null,"abstract":"<p><p>Chronic myelogenous leukemia at blast crisis with a T-cell phenotype (T-ALL CML-BC) at diagnosis, without any prior history of CML is extremely rare. After the introduction of tyrosine kinase inhibitors (TKIs), CML patients have a median survival comparable to general population and accelerated/blast crisis are rarely encountered. Most CML patients (80%) transform into acute myeloid leukemia and the rest into B-ALL. Anecdotal cases of Ph+ T-ALL, either de novo or in the context of CML-BC have been reported. Left shift in the blood, the presence of splenomegaly/extramedullary infiltration and the occurrence of BCR::ABL1 rearrangement in both the blastic population, as well as in the myeloid cell compartment are key points in differentiating de novo Ph+ T-ALL from T-ALL CML-BC. The latter is a rare entity, characterized by extramedullary disease, p210 transcript and clonal evolution. Lack of preceding CML does not rule out the diagnosis of T-ALL CML-BC. Prompt TKI treatment with ALL-directed therapy followed by allogeneic stem cell transplantation may offer long-term survival in this otherwise poor prognosis entity. In this paper, we describe a patient with T-ALL CML-BC at presentation, still alive 51 months after diagnosis and we offer a review of the literature on this rare subject. All clinical and laboratory features are provided in order to distinguish de novo Ph+ T-ALL from T-ALL CML-BC, underscoring the prognostic and therapeutic significance of such a differentiation.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"531-539"},"PeriodicalIF":1.7,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9966673","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"\"The Long Journey of Unexplained Erythrocytosis\": Erythrocytosis due to High-Oxygen Affinity Hemoglobinopathy - Hemoglobin Variant Little Rock (HBB: c.432C&gt;A) - A Report of a Swiss Family and Review of the Literature.","authors":"Camille Perroud, Naomi Porret, Alicia Rovó","doi":"10.1159/000530240","DOIUrl":"10.1159/000530240","url":null,"abstract":"<p><p>The differential diagnosis of erythrocytosis is complex, involving a tailored algorithm. Congenital causes are rare and such patients commonly face a long journey looking for diagnosis. This diagnosis requires expertise and accessibility to modern diagnostic tools. We present the case of a young Swiss man with long-standing erythrocytosis of unknown origin and his family. The patient had an episode of malaise as he went skiing above 2,000 m altitude. In the blood gas analysis, p50 was low (16 mm Hg) and erythropoietin was normal. Using next-generation sequencing, a mutation in the hemoglobin subunit beta gene was found, a pathogenic variant known as hemoglobin Little Rock causing high oxygen affinity. Some family members also had unexplained erythrocytosis, therefore the mutational status of the family was analyzed, the grandmother and mother showed the presence of the same mutation. The use of modern technology finally offered a diagnosis to this family.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 4","pages":"326-330"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9981556","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"False-Negative Testing for FIP1L1::PDGFRA by Fluorescence in situ Hybridization Is a Frequent Cause of Diagnostic Delay.","authors":"Thanai Pongdee, Alexis Berry, Lauren Wetzler, Xiaoping Sun, Lauren Thumm, Pryscilla Yoon, Fei Li Kuang, Michelle Makiya, Gregory Constantine, Paneez Khoury, Esther Rheinbay, Andrew A Lane, Irina Maric, Amy D Klion","doi":"10.1159/000528046","DOIUrl":"10.1159/000528046","url":null,"abstract":"<p><p>The imatinib-sensitive fusion gene FIP1L1::PDGFRA is the most frequent molecular abnormality identified in patients with eosinophilic myeloid neoplasms. Rapid recognition of this mutation is essential given the poor prognosis of PDGFRA-associated myeloid neoplasms prior to the availability of imatinib therapy. We report a case of a patient in whom delayed diagnosis resulted in cardiac transplantation for eosinophilic endomyocardial fibrosis. The delay in diagnosis was due, in part, to a false-negative result in fluorescence in situ hybridization (FISH) testing for FIP1L1::PDGFRA. To explore this further, we examined our cohort of patients presenting with confirmed or suspected eosinophilic myeloid neoplasms and found 8 additional patients with negative FISH results despite a positive reverse-transcriptase polymerase chain reaction test for FIP1L1::PDGFRA. More importantly, false-negative FISH results delayed the median time to imatinib treatment by 257 days. These data emphasize the importance of empiric imatinib therapy in patients with clinical features suggestive of PDGFRA-associated disease.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 4","pages":"316-321"},"PeriodicalIF":1.7,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10809802/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9986988","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment Outcomes of Acute Myeloid Leukemia in Patients Living with Human Immunodeficiency Virus Receiving Antiretroviral Therapy: A Single-Center Experience.","authors":"Jose Tinajero, Dat Ngo, Alfredo Puing, Paul Koller","doi":"10.1159/000533346","DOIUrl":"10.1159/000533346","url":null,"abstract":"<p><p>Patients living with HIV are now living longer due to increased access to antiretroviral therapy (ART) and a decrease in acquired immunodeficiency syndrome-defining cancer (ADC). However, increasing age and previous chemotherapy exposure for ADC (e.g., anthracyclines and topoisomerase inhibitors) are factors that may increase the risk of developing therapy-related myelodysplastic syndrome and acute myeloid leukemia (AML) and highlight an unmet need. There are no established guidelines for the treatment of AML in patients with HIV and the literature is limited to treatment outcomes and experience. In addition, cladribine, a purine analog used in AML, has a package insert warning to avoid administration with concurrent agents that undergo phosphorylation, which include HIV ART backbones (e.g., nucleoside reverse transcriptase inhibitors [NRTI]). Whether concurrent NRTI-based ART is deliverable with AML induction chemotherapy has not been reported previously. In our single-center experience of seven HIV-AML patients, all patients continued concurrent ART with induction chemotherapy. In 6 evaluable patients, three (50%) of patients went into complete remission (CR). Five (71.4%) patients were able to proceed to allogenic hematopoietic stem cell transplantation (HCT). Median OS was 16.6 months, with patients who received HCT having longer median OS compared to those who were unable to proceed to HCT (49.6 months vs. 3.4 months). Interestingly, none of the patients who received AML regimens that included fludarabine were able to obtain a response. On the contrary, 4 patients who received AML regimens that utilized cytarabine given over a prolonged period of time (e.g., 7 + 3, liposomal daunorubicin/cytarabine) achieved a CR rate of 75%. Concurrent HIV ART and AML induction chemotherapy is deliverable, although much remains to be investigated on potential drug interactions between purine analog-based chemotherapy and HIV ART.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"1 1","pages":"491-496"},"PeriodicalIF":1.7,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47735803","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Disparity Analysis of Clinical Features and Prognostic Evaluation of Hemophagocytic Lymphohistiocytosis in Pediatric and Adult Patients: A Retrospective Multicenter Study of 525 Cases in HHLWG.","authors":"Xiangmin Wang,&nbsp;Yi Zhou,&nbsp;Qian Sun,&nbsp;Hongyuan Zhou,&nbsp;Qing Zhang,&nbsp;Ziyuan Shen,&nbsp;Jie Huang,&nbsp;Yao Xue,&nbsp;Dongmei Yan,&nbsp;Yongjun Fang,&nbsp;Wei Sang","doi":"10.1159/000528934","DOIUrl":"https://doi.org/10.1159/000528934","url":null,"abstract":"<p><strong>Introduction: </strong>Hemophagocytic lymphohistiocytosis (HLH) is a rare immune disorder with rapid progression and high mortality. There have been few large cohort study comparisons of pediatric and adult HLH until now. This study was designed to explore the disparity of clinical presentations and evaluate the prognosis in pediatric and adult HLH patients.</p><p><strong>Methods: </strong>Totally, 525 newly diagnosed HLH patients were included and divided into 4 groups according to age: <6, 6-18, 18-60, and >60 years (geriatric patients). Mann-Whitney U test, Kruskal-Wallis test, χ2 test, and Bonferroni's adjustment were used to explore the difference between age groups. Overall survival (OS) was estimated by using Kaplan-Meier method. The Cox proportional hazard model was used to analyze the univariable and multivariable association between prognostic factors and OS.</p><p><strong>Results: </strong>Geriatric patients had the lowest levels of hemoglobin, platelet, albumin, and the highest level of creatinine, while patients <6 years of age had the lowest values of fibrinogen, IgA, IgM and highest values of triglyceride. The trigger of HLH in patients <18 years of age was mainly EBV infection. However, lymphoma and non-EBV-driven infection were the more frequent drivers in patients aged 18-60 and >60 years, respectively. Geriatric patients were associated with highest mortality (58.8%), and 5-year OS was 43%. By contrast, 5-year OS of patients <6, 6-18, and 18-60 years was 86.1%, 74%, and 58.9%, respectively. Additionally, among patients with different etiologies (EBV, non-EBV-driven infection, and uncertain causes) and treatment regimens (HLH-04, HLH-94, and glucocorticoid regimen), geriatric patients showed lowest 5-year OS. Multivariate analysis revealed that creatinine and alanine aminotransferase were independent risk factors affecting the survival of patients aged 0-6 years, while albumin and IgG were independent factors affecting survival of geriatric patients.</p><p><strong>Conclusion: </strong>Our study showed a wide heterogeneity of clinical presentations, etiology distribution, prognostic factors, and survival outcomes in pediatric and adult HLH patients.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 3","pages":"185-195"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9573926","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acquired Von Willebrand Syndrome following a SARS-CoV2 Infection.","authors":"Michael Iarossi,&nbsp;Cedric Hermans","doi":"10.1159/000529740","DOIUrl":"https://doi.org/10.1159/000529740","url":null,"abstract":"<p><p>Acquired von Willebrand syndrome is a rare clinical entity with approximately 700 cases described in the literature. Different etiologies can be responsible for the occurrence of this condition, including mainly lymphoproliferative and myeloproliferative syndromes, as well as cardiac diseases. Several mechanisms have been implicated depending on the etiology. Viral infections are an extremely rare cause, with only one case reported after an Epstein-Barr virus infection. In this case report, we have described the very likely association between SARS-CoV2 infection and the development of a time-limited acquired von Willebrand syndrome.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"146 3","pages":"226-229"},"PeriodicalIF":2.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10090405/pdf/aha-0001.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9573971","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}