1372 results found
Cross NCP, Ernst T, Branford S, et al., 2023, European LeukemiaNet laboratory recommendations for the diagnosis and management of chronic myeloid leukemia, Leukemia, Vol: 37, Pages: 2150-2167, ISSN: 0887-6924
From the laboratory perspective, effective management of patients with chronic myeloid leukemia (CML) requires accurate diagnosis, assessment of prognostic markers, sequential assessment of levels of residual disease and investigation of possible reasons for resistance, relapse or progression. Our scientific and clinical knowledge underpinning these requirements continues to evolve, as do laboratory methods and technologies. The European LeukemiaNet convened an expert panel to critically consider the current status of genetic laboratory approaches to help diagnose and manage CML patients. Our recommendations focus on current best practice and highlight the strengths and pitfalls of commonly used laboratory tests.
Ortí G, Gras L, Koster L, et al., 2023, Graft-versus-Host Disease Prophylaxis with Post- Transplantation Cyclophosphamide in Chronic Myeloid Leukemia Patients Undergoing Allogeneic Hematopoietic Cell Transplantation from an Unrelated or Mismatched Related Donor: A Comparative Study from the Chronic Malignancies Working Party of the EBMT (CMWP-EBMT)., Transplant Cell Ther
Outcomes following allogeneic hematopoietic cell transplantation (allo-HCT) for chronic myeloid leukemia (CML) with post-transplantation cyclophosphamide (PTCy) using an unrelated donor (UD) or a mismatched related donor (MMRD) remain unknown. We report a retrospective comparison of PTCy-based allo-HCT from a UD, non-PTCy allo-HCT from a UD, and PTCy allo-HCT from an MMRD. Inclusion criteria were adult patients with CML undergoing first allo-HCT between 2012 and 2019 from a UD with either PTCy or non-PTCy graft-versus-host disease (GVHD) prophylaxis or from an MMRD using PTCy. The primary endpoint was GVHD-free/relapse-free survival (GRFS). A total of 1341 patients were included (82% in the non-PTCy UD cohort). With a median follow-up of 34.9 months, the 3-year GRFS was 43% in the non-PTCy cohort, 37% in the PTCy-UD cohort, and 39% PTCy-MMRD cohort (P = .15). Multivariable analyses revealed no significant differences among the 3 cohorts in terms of overall survival (OS), progression-free survival, RI, and nonrelapse mortality. Factors independently associated with worse OS in the overall cohort were Karnofsky Performance Status <90 (hazard ratio [HR], 1.86; 95% confidence interval [CI], 1.41 to 2.45; P < .001), older age (HR, 1.24, 95% CI, 1.11 to 1.38; P < .001), and disease stage (compared to chronic phase [CP] 1): blast phase (HR, 2.25; 95% CI, 1.60 to 3.16; P < .001), accelerated phase (HR, 1.63; 95% CI, 1.05 to 2.54; P = .03), and CP >2 (HR, 1.58; 95% CI, 1.15 to 2.17; P = .005). These results suggest that allo-HCT in patients with CML using either a UD or an MMRD with PTCy-based GVHD prophylaxis are feasible transplantation, platforms and that the disease stage at allo-HCT remains a major prognostic factor, highlighting the importance of closely monitoring CML patients and proposing transplantation when indicated when still in CP1.
Mauro M, Hochhaus A, Hughes T, et al., 2023, Responses With Asciminib Continue to Deepen Over Time in Patients With Chronic Myeloid Leukemia in Chronic Phase (CML-CP) After ≥2 Prior Tyrosine Kinase Inhibitors (TKIs) in the Phase III ASCEMBL Study, Publisher: CIG MEDIA GROUP, LP, Pages: S340-S340, ISSN: 2152-2650
Apperley J, Cortes J, Jabbour E, et al., 2023, Molecular Response of ≤10% BCR::ABL1IS Is Predictive of Positive Outcomes in Patients With Treatment-Resistant Chronic-Phase Chronic Myeloid Leukemia (CP-CML) Treated With Ponatinib From the Phase 2 OPTIC Trial, Publisher: CIG MEDIA GROUP, LP, Pages: S339-S339, ISSN: 2152-2650
Cortes J, Deininger M, Lomaia E, et al., 2023, Post Hoc Analysis of Patient Responses by T315I Mutation Status From the 3-Year Update of the OPTIC Trial: A Dose-Optimization Study of 3 Starting Doses of Ponatinib, Publisher: CIG MEDIA GROUP, LP, Pages: S338-S338, ISSN: 2152-2650
Robin M, Gras L, Koster L, et al., 2023, Are syngeneic donors a viable donor option in allogeneic haematopoietic cell transplantation for MDS? A brief report on behalf of the Chronic Malignancies Working Party of the EBMT and review of current literature, BONE MARROW TRANSPLANTATION, Vol: 58, Pages: 942-945, ISSN: 0268-3369
May P, Reid A, Robinson M, et al., 2023, FISH-negative BCR::ABL1-positive e19a2 chronic myeloid leukaemia: the most cryptic of insertions, BMC Medical Genomics, Vol: 16, Pages: 1-6, ISSN: 1755-8794
Background:Chronic myeloid leukaemia (CML) is one of the most well characterised human malignancies. Most patients have a cytogenetically visible translocation between chromosomes 9 and 22 which generates the pathognomonic BCR::ABL1 fusion gene. The derivative chromosome 22 (‘Philadelphia’ or Ph chromosome) usually harbours the fusion gene encoding a constitutively active ABL1 kinase domain. A small subset of patients have no detectable translocation. Historically, these ‘Philadelphia chromosome negative’ patients caused diagnostic confusion between CML and other myeloproliferative neoplasms; it is now well established that the BCR::ABL1 fusion gene can be generated via submicroscopic intrachromosomal insertion of ABL1 sequence into BCR, or, more rarely, of BCR into ABL1. The fusion genes arising from cryptic insertions are not detectable via G-banded chromosome analysis [karyotype] but can nevertheless always be detected using fluorescence in situ hybridisation (FISH) and/or qualitative reverse transcriptase PCR.Case presentation:A 43-year-old female presented with suspected CML in 2007; however, contemporaneous gold standard laboratory investigations, G-banded chromosome analysis and FISH, were both negative. The reverse transcriptase quantitative PCR (RT-qPCR) assay available at the time, which was capable of detecting the common BCR::ABL1 transcripts (e13a2/e14a2), was also negative. Upon review in 2009, the newly recommended reverse transcriptase multiplex PCR (capable of detecting all BCR::ABL1 transcripts including the atypical ones) subsequently detected an e19a2 fusion. The patient then responded to tyrosine kinase inhibitor therapy. In contrast, FISH studies of both samples with three commercially available probes remained consistently negative.Retrospective whole genome sequencing, undertaken as part of the 100,000 Genomes Project, has now revealed that the patient’s BCR::ABL1 fusion gene arose via a uniquely small insertion
Fernando F, Innes AJ, Claudiani S, et al., 2023, The outcome of post-transplant asciminib in patients with chronic myeloid leukaemia, BONE MARROW TRANSPLANTATION, Vol: 58, Pages: 826-828, ISSN: 0268-3369
Sweet KL, Cortes JE, Apperley JF, et al., 2023, Project Confirm: Accelerated Drug Approvals for Chronic Myeloid Leukemia, CLINICAL CANCER RESEARCH, Vol: 29, Pages: 2179-2183, ISSN: 1078-0432
Robertson HF, Buckton MJ, Apperley JF, 2023, Managing women of childbearing age with chronic myeloid leukemia: safety and treatment considerations, EXPERT REVIEW OF HEMATOLOGY, Vol: 16, Pages: 325-332, ISSN: 1747-4086
Read C, Apperley JF, Hettiaratchy SP, et al., 2023, The impact of mental health comorbidities on patient satisfaction: A population study among US adults with skin cancer, 1st International Societies for Investigative Dermatology Meeting, Publisher: ELSEVIER SCIENCE INC, Pages: S77-S77, ISSN: 0022-202X
Nesr G, Braithwaite B, Mathew P, et al., 2023, Generic imatinib: Data from the UK National Registry for chronic myeloid leukaemia patients, 63rd Annual Scientific Meeting of the British-Society-for-Haematology, Publisher: WILEY, Pages: 92-93, ISSN: 0007-1048
Hochhaus A, Rea D, Boquimpani C, et al., 2023, Asciminib vs bosutinib in chronic-phase chronic myeloid leukemia previously treated with at least two tyrosine kinase inhibitors: longer-term follow-up of ASCEMBL, LEUKEMIA, Vol: 37, Pages: 617-626, ISSN: 0887-6924
Ruggeri A, De Wreede LC, Müller CR, et al., 2023, Integrating biological HLA-DPB1 mismatch models to predict survival after unrelated hematopoietic cell transplantation., Haematologica, Vol: 108, Pages: 645-652
Milojkovic D, Reynolds CJ, Sandoval DM, et al., 2023, COVID-19 vaccine boosted immunity against Omicron in chronic myeloid leukemia patients treated with tyrosine kinase inhibitors, LEUKEMIA, Vol: 37, Pages: 244-247, ISSN: 0887-6924
Chalandon Y, Sbianchi G, Gras L, et al., 2023, Allogeneic hematopoietic cell transplantation in patients with chronic phase chronic myeloid leukemia in the era of third generation tyrosine kinase inhibitors: A retrospective study by the chronic malignancies working party of the EBMT, AMERICAN JOURNAL OF HEMATOLOGY, Vol: 98, Pages: 112-121, ISSN: 0361-8609
Robertson HF, Apperley JF, 2022, Treatment of CML in pregnancy, HEMATOLOGY-AMERICAN SOCIETY OF HEMATOLOGY EDUCATION PROGRAM, Pages: 123-128, ISSN: 1520-4391
Branford S, Apperley JF, 2022, Measurable residual disease in chronic myeloid leukemia, HAEMATOLOGICA, Vol: 107, Pages: 2794-2809, ISSN: 0390-6078
Cortes JE, Deininger MW, Lomaia E, et al., 2022, Three-Year Update from the Optic Trial: A Dose-Optimization Study of 3 Starting Doses of Ponatinib, 64th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH), Publisher: AMER SOC HEMATOLOGY, Pages: 1495-1497, ISSN: 0006-4971
Apperley JF, Cortes JE, Jabbour E, et al., 2022, Molecular Response of ≤10% <i>BCR::ABL1</i><SUP>IS</SUP> Is Predictive of Positive Outcomes in Treatment-Resistant Patients with Chronic Phase Chronic Myeloid Leukemia (CP-CML) Treated with Ponatinib from the Phase 2 Optic Trial, 64th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH), Publisher: AMER SOC HEMATOLOGY, Pages: 6760-6762, ISSN: 0006-4971
Dominy K, Salmon M, Szydlo R, et al., 2022, Digital Droplet (dd) Polymerase Chain Reaction (PCR) Assays Offer Limited Advantages over Conventional Reverse-Transcriptase Quantitative PCR (RT-qPCR) for the Prediction of Molecular Recurrence after Treatment Discontinuation in Chronic Myeloid Leukemia (CML): Results from the Destiny Study, 64th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH), Publisher: AMER SOC HEMATOLOGY, Pages: 9631-9632, ISSN: 0006-4971
Chelysheva EY, Turkina A, Apperley JF, et al., 2022, Chronic Myeloid Leukemia Diagnosed during Pregnancy: How to Manage? Description of 86 Cases from ELN International Registry, 64th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH), Publisher: AMER SOC HEMATOLOGY, Pages: 1498-1500, ISSN: 0006-4971
Cortes JE, Saikia T, Kim D-W, et al., 2022, Efficacy and Safety of Vodobatinib in Patients (pts) with Chronic Phase Philadelphia Positive Chronic Myeloid Leukemia (Ph plus CML): A Sub Group Analysis By Lines of Tyrosine Kinase Inhibitor (TKI) Therapy, 64th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH), Publisher: AMER SOC HEMATOLOGY, ISSN: 0006-4971
Hughes T, Rea D, Boquimpani C, et al., 2022, Dynamics of Response and Response Factors in Patients (Pts) with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) after ≥2 Prior Tyrosine Kinase Inhibitors (TKIs) in the Phase 3 Ascembl Study, 64th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH), Publisher: AMER SOC HEMATOLOGY, Pages: 6757-6759, ISSN: 0006-4971
Innes AJ, Hayden C, Orovboni V, et al., 2022, Real-World Experience of Asciminib: Factors Associated with Response, 64th Annual Meeting and Exposition of the American-Society-of-Hematology (ASH), Publisher: AMER SOC HEMATOLOGY, Pages: 6796-6797, ISSN: 0006-4971
Cook L, O'dell G, Vourvou E, et al., 2022, Third primary SARS-CoV-2 mRNA vaccines enhance antibody responses in most patients with haematological malignancies, Nature Communications, Vol: 13, Pages: 1-6, ISSN: 2041-1723
SARS-CoV-2 infection, and resulting disease, COVID-19, has a high mortality amongst patients with haematological malignancies. Global vaccine rollouts have reduced hospitalisations and deaths, but vaccine efficacy in patients with haematological malignancies is known to be reduced. The UK-strategyoffered a third, mRNA-based, vaccine as an extension to the primary course in these patients. The MARCH database is a retrospective observational study of serological responses in patients with blood disorders. Here we present data on 381 patients with haematological malignancies. By comparison with healthy controls, we report suboptimal responses following two primary vaccines, with significantly enhanced responses following the third primary dose. These responses however are heterogeneous and determined by haematological malignancy sub-type and therapy. We identify a group of patients with continued sub-optimal vaccine responses who may benefit from additional doses, prophylactic extended half-life neutralising monoclonal therapies (nMAB) or prompt nMAB treatment in the event of SARS-CoV-2 infection.
Saraceni F, Labopin M, Angelucci E, et al., 2022, Treosulfan-based compared to thiotepa-busulfan-fludarabine conditioning for haploidentical transplant in patients with acute myeloid leukemia. A study from the Acute Leukemia Working Party of the EBMT, 48th Annual Meeting of the European-Society-for-Blood-and-Marrow-Transplantation (EBMT), Publisher: SPRINGERNATURE, Pages: 36-37, ISSN: 0268-3369
Kantarjian HM, Jabbour E, Deininger M, et al., 2022, Ponatinib after failure of second-generation tyrosine kinase inhibitor in resistant chronic-phase chronic myeloid leukemia, AMERICAN JOURNAL OF HEMATOLOGY, Vol: 97, Pages: 1419-1426, ISSN: 0361-8609
Albert MH, Sirait T, Eikema D-J, et al., 2022, Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study., Blood, Vol: 140, Pages: 1635-1649
Allogeneic hematopoietic stem cell transplantation (HSCT) is the gold standard curative therapy for infants and children with many inborn errors of immunity (IEI), but adolescents and adults with IEI are rarely referred for transplant. Lack of published HSCT outcome data outside small, single-center studies and perceived high risk of transplant-related mortality have delayed the adoption of HSCT for IEI patients presenting or developing significant organ damage later in life. This large retrospective, multicenter HSCT outcome study reports on 329 IEI patients (age range, 15-62.5 years at HSCT). Patients underwent first HSCT between 2000 and 2019. Primary endpoints were overall survival (OS) and event-free survival (EFS). We also evaluated the influence of IEI-subgroup and IEI-specific risk factors at HSCT, including infections, bronchiectasis, colitis, malignancy, inflammatory lung disease, splenectomy, hepatic dysfunction, and systemic immunosuppression. At a median follow-up of 44.3 months, the estimated OS at 1 and 5 years post-HSCT for all patients was 78% and 71%, and EFS was 65% and 62%, respectively, with low rates of severe acute (8%) or extensive chronic (7%) graft-versus-host disease. On univariate analysis, OS and EFS were inferior in patients with primary antibody deficiency, bronchiectasis, prior splenectomy, hepatic comorbidity, and higher hematopoietic cell transplant comorbidity index scores. On multivariable analysis, EFS was inferior in those with a higher number of IEI-associated complications. Neither age nor donor had a significant effect on OS or EFS. We have identified age-independent risk factors for adverse outcome, providing much needed evidence to identify which patients are most likely to benefit from HSCT.
Apperley J, Kantarjian H, Deininger M, et al., 2022, Dose Modification Dynamics of Ponatinib in Patients With Chronic-Phase Chronic Myeloid Leukemia From the PACE and OPTIC Trials, CLINICAL LYMPHOMA MYELOMA & LEUKEMIA, Vol: 22, Pages: S284-S285, ISSN: 2152-2650
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