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ALL (Acute Lymphoblastic Leukemia)Other CancersBiological: BlinatumomabBiological: RituximabCyclophosphamideCytarabineDaunorubicinDaunorubicin HydrochlorideDexamethasoneEtoposideMercaptopurineMethotrexatePegaspargasePrednisoneProcedure: Allogeneic Hematopoietic Stem Cell TransplantationProcedure: Biospecimen CollectionProcedure: Bone Marrow AspirationProcedure: Bone Marrow BiopsyProcedure: Lumbar PunctureProcedure: X-Ray ImagingVincristineVincristine SulfateActiveNot Yet RecruitingCanadaIsraelPuerto RicoUnited States 0NCT02003222Phase 3
Combination Chemotherapy With or Without Blinatumomab in Treating Patients With Newly Diagnosed BCR-ABL-Negative B Lineage Acute Lymphoblastic Leukemia

Combination Chemotherapy With or Without Blinatumomab in Treating Patients With Newly Diagnosed BCR-ABL-Negative B Lineage Acute Lymphoblastic Leukemia

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Eribulin and Nivolumab Combo for advanced breast cancer
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Prevalence and Outcomes of gBRCAm in Early-Stage BC

KEY TAKEAWAYS The study aimed to investigate the prevalence and outcomes of pathogenic BRCA1/2 variants in patients with early-stage BC. Researchers noticed that similar survival outcomes among gBRCAm and gBRCAwt patients indicate a need for broader screening...

Sep 27, 2024

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Real-World Acalabrutinib: High RW-PFS & Continuation Rates

KEY TAKEAWAYS

  • The EPIC trial aimed to present interim analysis of outcomes, patient characteristics, and treatment patterns in CLL post-acalabrutinib initiation.
  • The interim analysis reported real-world acalabrutinib outcomes aligning with ELEVATE-TN trial findings, informing UK clinical decisions.

High response rates and acceptable safety profiles were noted in clinical trials of acalabrutinib, a second-generation Bruton’s tyrosine kinase inhibitor, for patients with chronic lymphocytic leukemia (CLL). Nonetheless, there is a scarcity of UK real-world data regarding the utilization of first-line acalabrutinib in patients with CLL.

Nicolas Martinez-Calle and the team aimed to deliver an interim analysis of outcomes, clinical features, and treatment trends in patients with CLL following the initiation of first-line acalabrutinib via the UK Early Access Programme (EAP), with a minimum follow-up of 24 months.

This retrospective multi-center cohort study, registered under ClinicalTrials.gov (NCT05557695), focused on patients with CLL eligible if treatment-naïve and beginning acalabrutinib via the EAP between April 1, 2020, and April 1, 2021.

Patients, recruited from 16 sites in England and Wales, were assessed for real-world progression-free survival (rwPFS), real-world overall survival (rwOS), and real-world overall response rate (rwORR) during first-line treatment.

Data included baseline clinical and demographic characteristics as well as acalabrutinib treatment patterns, sourced from medical records. The study intends to track patients for up to 60 months post-acalabrutinib initiation (index date), with a data cut-off on November 30, 2023.

The results revealed that this interim analysis comprised 170 patients, with a median age at index of 74.5 years (IQR, 68.3–79.6). Among them, 41% were female, and the majority were White British. Most patients had an Eastern Cooperative Oncology Group performance score of 0-1.

The median Cumulative Illness Rating Scale-Geriatric score at index was 4. Acalabrutinib was primarily administered at a dose of 100 mg bi-daily. The median duration of follow-up was 36.9 months. rwPFS rate at 12 and 24 months was 92.4% (95% CI, 88.4 – 96.4) and 87.0% (95% CI, 82.1 – 92.2), respectively. The rwOS rate at 12 and 24 months was 92.9% (95% CI, 89.2–96.9) and 88.2% (95% CI, 83.5–93.2), respectively. The rwORR at 12 and 24 months was 87.0% (95% CI, 80.6–93.7; n=101) and 86.0% (95% CI, 79.9–92.0; n=128), respectively.

At index, a notable proportion of patients had creatinine clearance <60 mL/min and various mutations were observed. The median time from CLL diagnosis to index was 3.2 years. A significant portion of patients had comorbidities such as hypertension, type-2 diabetes, kidney disease, and atrial fibrillation.

Treatment continuation rates at 12 and 24 months were 87.6% (95% CI, 82.7–92.7, n=169) and 79.3% (95% CI,73.4–85.6, n=169), respectively. At data cut-off, 24% (40/169) patients had discontinued acalabrutinib (1 had no record); 38% (15/40) of discontinuations were due to adverse events, 13% (5/40) disease progression, 5% (2/40) patients’ decision and 43% (17/40) other (1 patient had no reason recorded); most common adverse events leading to discontinuations (≥1 AE could be reported per discontinuation) included cardiovascular, haematoma, and Richter transformation (n=2 each).

The study concluded that real-world acalabrutinib outcomes, including a 24-month rwPFS rate of 87.0% and a treatment continuation rate of 79.3%, align with findings from the ELEVATE-TN trial (NCT02475681). These results offer valuable insights for clinical decision-makers regarding the utilization of acalabrutinib in UK settings.

The trial was sponsored by AstraZeneca.

Source: https://library.ehaweb.org/eha/2024/eha2024-congress/419920/nicolas.martinez-calle.outcomes.of.epic.a.non-interventional.observational.html?f=menu%3D6%2Abrowseby%3D8%2Asortby%3D2%2Ace_id%3D2552%2Aot_id%3D29194%2Amarker%3D5100%2Afeatured%3D18527

Clinical Trial: https://clinicaltrials.gov/study/NCT05557695

Martinez-Calle N, Walewska R, Paneesha S, et al. (2024). “OUTCOMES OF EPIC: A NON-INTERVENTIONAL, OBSERVATIONAL STUDY OF PATIENTS WITH CHRONIC LYMPHOCYTIC LEUKAEMIA TREATED WITH FIRST-LINE ACALABRUTINIB VIA THE UK EARLY ACCESS PROGRAMME; INTERIM ANALYSES.” Presented at EHA 2024 (P1833).

Efficient MRD Assessment with Dr. PCR in CML

KEY TAKEAWAYS

  • The study aimed to assess Dr. PCR’s accuracy in quantifying BCR::ABL1 transcripts for MRD monitoring in patients with CML.
  • Researchers concluded that Dr. PCR offers rapid and efficient MRD assessment.

Accurate quantification of the BCR::ABL1 transcripts is crucial for measurable residual disease (MRD) monitoring in chronic myeloid leukemia (CML) following tyrosine kinase inhibitor (TKI) treatment.

Soo Jung Lee and the team aimed to evaluate the newly developed digital real-time PCR method, Dr. PCR, as an alternative to reverse transcription-PCR (qRT-PCR) for MRD detection.

They conducted a comprehensive analysis, evaluating Dr. PCR’s performance using both reference and clinical materials. They assessed precision, linearity, and correlation with qRT-PCR. They compared MRD levels detected by Dr. PCR with qRT-PCR and investigated practical advantages.

Dr. PCR detected MRD up to 0.0032%IS (MR4.5) with excellent precision and linearity and showed a strong correlation with qRT-PCR results. Notably, Dr. PCR identified higher levels of MRD in 12.7% (29/229) of patients than qRT-PCR, including 6 cases of MR4, which is a critical level for TKI discontinuation. Dr. PCR also allowed for sufficient ABL1 copies in all cases, while qRT-PCR necessitated multiple repeat tests in 3.5% (8/229) of cases.

The study concluded that Dr. PCR offers a rapid and efficient method for assessing MRD in patients with CML under the current treatment regimen, supported by a robust body of evidence.

This study was supported by The Catholic University of Korea.

Source: https://pubmed.ncbi.nlm.nih.gov/38525919/

Lee SJ, Lee JM, Ahn A, et al.(2024). “Analytical Performance Evaluation of a Digital Real-Time PCR for Quantifying Major BCR::ABL1 Transcripts.” J Clin Lab Anal. 2024 Apr;38(7):e25034. doi: 10.1002/jcla.25034. Epub 2024 Mar 25. PMID: 38525919; PMCID: PMC11033343.

Ibrutinib Induces Atrial Arrhythmia via PI3K Pathway

KEY TAKEAWAYS

  • The study aimed to understand the precise mechanism behind ibrutinib-induced atrial fibrillation in patients with CLL.
  • The results revealed that the ibrutinib’s off-target effect on PI3K-AKT-mTOR causes connexin degradation, promoting autophagy and atrial arrhythmia.

Ibrutinib has been associated with an increased risk of atrial fibrillation (AF) in patients with chronic lymphocytic leukemia (CLL). However, the precise mechanism behind this effect is not yet fully understood.

Huiyuan Qin and the team aimed to determine the incidence of new-onset atrial fibrillation (AF) in patients with CLL receiving ibrutinib and elucidate its underlying mechanism.

Researchers investigated the prevalence of new-onset AF in patients with CLL undergoing treatment with ibrutinib. They utilized optical mapping to elucidate the proarrhythmic effects of ibrutinib on HL-1 cells.

Furthermore, fluorescence staining and western blot analysis were employed to compare the expression levels of connexins 43 and 40 between the ibrutinib-treated group and the control group. To characterize autophagy phenotypes, they utilized western blotting to assess the levels of autophagy-related proteins.

The study employed transmission electron microscopy (TEM) to visualize autophagosomes and used a transfected mCherry-GFP-LC3 virus to label autophagosomes and lysosomes. Additionally, hydroxychloroquine, an autophagy inhibitor, was administered to counteract ibrutinib-induced degradation of Cx43 and Cx40.

About 2.67% of patients experienced atrial arrhythmias following the administration of ibrutinib. HL-1 cells subjected to ibrutinib treatment displayed decreased conduction velocity and a heightened occurrence of reentry-like arrhythmias compared to the control group.

Furthermore, the expression of connexins 43 and 40 was reduced, accompanied by an increase in autophagy markers in HL-1 cells treated with ibrutinib. The inhibition of autophagy led to the upregulation of connexins 43 and 40.

The off-target impact of ibrutinib on the PI3K-AKT-mTOR signaling pathway resulted in the degradation of connexins and the onset of atrial arrhythmia by inducing autophagy.

Funding was provided by the National Nature Science Foundation of China.

Source: https://pubmed.ncbi.nlm.nih.gov/38812314/

Qin H, Zheng B, Lin Z, et al. (2024). “Ibrutinib Contributes to Atrial Arrhythmia through the Autophagic Degradation of Connexins by Inhibiting the PI3K-AKT-mTOR Signaling Pathway.” Front Biosci (Landmark Ed). 2024 May 22;29(5):201. doi: 10.31083/j.fbl2905201. PMID: 38812314.

Pharmacodynamic Parameters and Ibrutinib Response in CLL

Pharmacodynamic Parameters and Ibrutinib Response in CLL

KEY TAKEAWAYS

  • The PK-E3I observational study aimed to investigate how transient vs pHL affects outcomes in patients with CLL treated with ibrutinib.
  • Researchers noted that tHL is linked to reduced response duration, indicating the need for therapy adjustment after 3 months.

One of the 1 clinical observations of ibrutinib activity in the treatment of chronic lymphocytic leukemia (CLL) is a rapid decline in lymph node size. This phenomenon is accompanied by hyperlymphocytosis, either transient or prolonged, which is associated with distinct clinical responses and thus impacts long-term outcomes. Understanding the factors that determine different disease courses upon ibrutinib treatment remains a scientific challenge.

Sarah Cadot and the team aimed to assess the effects of transient versus prolonged hyperlymphocytosis (pHL) on clinical outcomes and pharmacodynamic parameters in patients with CLL undergoing ibrutinib treatment.

They performed an inclusive analysis from 2016 to 2021 with 2 cohorts of patients with CLL (cohort 1, n = 41; cohort 2, n = 81), reflecting the typical clinical characteristics of CLL. Such as a Male/Female sex ratio of 2/1, a median age of 70 years at diagnosis, and patients in first-line (1L) therapy (27%) or relapsed/refractory (R/R) patients (73%).

The study tracked blood cell counts over 2 years of ibrutinib treatment and included immunophenotyping and whole-body magnetic resonance imaging (MRI) in cohort 1. Data were integrated into a newly developed mathematical model, combining dynamical and statistical approaches, to identify biological mechanisms associated with different clinical responses.

The analysis revealed that ibrutinib-induced lymphocytosis delineates 2 patients with CLL subgroups: transient hyperlymphocytosis (tHL) and pHL.

Differences between the two groups were determined by baseline parameters such as absolute counts of CD4+ T lymphocytes (P = 0.026), regulatory CD4 T cells (P = 0.007), programmed cell death protein 1 (PD1), and CD69 expression on B leukemic cells, CD19/CD5high/CXCR4low levels (P = 0.04), and lymph node cellularity.

The study found that patients with tHL had a lower duration of response and poorer clinical outcomes. The mathematical modeling reproduced patient-specific dynamics and highlighted that differences between the 2 groups were largely due to the production of leukemic B cells in lymph node compartments and, to a lesser extent, T lymphocytes and leukemic B cell egress into the bloodstream.

Access to additional longitudinal MRI data could further elucidate leukemic B cell dynamics in lymph nodes and validate the relevance of the 2 distinct patient groups.

The study concluded that the multidisciplinary approach improved the understanding of ibrutinib response by identifying new pharmacodynamic parameters before and during treatment. It highlighted that tHL is associated with reduced duration of response and poorer clinical outcomes. Consequently, the findings support the need for adjusted management of ibrutinib therapy after 3 months.

The study was funded by Institut National du Cancer within the framework of C15093BS-Cancer Plan (SC, SK, LD, LY, AQM); Agence Nationale de la Recherche within the framework of LABEX-TOUCAN (SC, CL, LY, AQM); Centre National de la Recherche Scientifique within the framework of EDYLE (SC, CA, CD, LD, LY, FC, AQM); La Ligue contre le Cancer, within the framework of Comites 11,32,65 (LY, AQM).

Source: https://pubmed.ncbi.nlm.nih.gov/39037964/

Clinical Trial: https://clinicaltrials.gov/study/NCT02824159

Cadot S, Audebert C, Dion C, et al. (2024). “New pharmacodynamic parameters linked with ibrutinib responses in chronic lymphocytic leukemia: Prospective study in real-world patients and mathematical modeling.” PLoS Med. 2024 Jul 22;21(7):e1004430. doi: 10.1371/journal.pmed.1004430. PMID: 39037964.

Targeted IGHV SHM in CLL for Prognosis Integration

KEY TAKEAWAYS

  • The study aimed to investigate the efficacy of a targeted capture approach for determining SHM status in the IGHV gene in patients with CLL.
  • Researchers noticed that a targeted capture approach enables broad CLL prognostication via IGHV SHM integration in a single assay.

The somatic hypermutation (SHM) status of the immunoglobulin heavy variable (IGHV) gene is pivotal for assessing the prognosis and guiding treatment decisions in patients with chronic lymphocytic leukemia (CLL). However, the conventional method for determining SHM status, involving multiplex polymerase chain reaction (PCR) and Sanger sequencing of the immunoglobulin heavy locus, suffers from limitations such as low throughput, susceptibility to failure, and lack of compatibility with multiplexing other diagnostic assays.

Jennifer M Grants and the team aimed to address these challenges by exploring an alternative approach for assessing SHM status in patients with CLL.

They conducted an inclusive analysis wherein a DNA-targeted capture approach was devised and validated to detect SHM status in the IGHV gene. This method was integrated as a submodule within a larger next-generation sequencing (NGS) panel comprising probes for various genes associated with CLL prognosis and treatment, including ATM, BIRC3, CHD2, KLHL6, MYD88, NOTCH1, NOTCH2, POT1, SF3B1, TP53, and XPO1. Inputting FASTQ files, the assay yields a comprehensive report consistent with the European Research Initiative on CLL guidelines, detailing IGHV SHM status and V allele usage.

The approach was validated on 35 CLL patient samples, 34 of which had been characterized via Sanger sequencing. Using the NGS panel, IGHV SHM status was successfully identified in 34 out of 35 patients with CLL. Sensitivity and specificity were demonstrated to be 100% among the 33 CLL samples with both NGS and Sanger sequencing calls. Additionally, it was shown that this panel could be integrated with other targeted capture panels to detect prognostically relevant CLL single nucleotide variants, insertions/deletions, and copy number variants, particularly TP53 copy number loss.

The study concluded that a targeted capture approach for detecting IGHV SHM can seamlessly integrate into broader sequencing panels, thereby enabling comprehensive prognostication of CLL within a single molecular assay.

The study was sponsored by a Large Scale Applied Research Project funded by Genome Canada, Genome British Columbia, Canadian Institutes of Health Research, the British Columbia Cancer Foundation, and the Provincial Health Services Authority.

Source: https://pubmed.ncbi.nlm.nih.gov/38175592/

Grants JM, May C, Bridgers J, et al. (2024). “Chronic Lymphocytic Leukemia IGHV Somatic Hypermutation Detection by Targeted Capture Next-Generation Sequencing.” Clin Chem. 2024 Jan 4;70(1):273-284. doi: 10.1093/clinchem/hvad147. PMID: 38175592.

JAK2 Levels as CML Treatment Response Indicator

KEY TAKEAWAYS

  • The study aimed to examine JAK2 levels’ association with CHR and early molecular response post-3-month imatinib treatment in chronic phase CML.
  • The results revealed that JAK2 levels could be a potential indicator for assessing imatinib treatment response in CML, given its involvement in the disease’s pathophysiology.

Chronic myeloid leukemia (CML), also known as chronic granulocytic leukemia, is characterized by the presence of the Philadelphia (Ph+) chromosome, indicating a myeloproliferative neoplasm. Imatinib, a first-line tyrosine kinase inhibitor, remains fundamental in treatment protocols. However, treatment resistance has been observed, partly attributed to the role of additional genes like Janus kinase 2 (JAK2).

Indra Wijaya and the team aimed to assess the correlation between JAK2 levels and complete hematological response (CHR) and early molecular response (EMR) post-3-month imatinib therapy in patients with chronic phase CML.

Patients diagnosed with Ph+ CML in the chronic phase (n = 40; mean age, 40 ± 11 years) were enrolled to undergo assessments, which included clinical examination and blood tests. These evaluations, including complete blood counts and JAK2 levels, were conducted at baseline and after 3 months of treatment with imatinib (at a daily oral dose of 400 mg). Participants were categorized into 2 groups based on the presence or absence of CHR and EMR.

The results revealed that in the group exhibiting CHR and EMR, levels of JAK2 gene, phosphorylated, and total JAK2 proteins were notably lower at baseline. Furthermore, a negative correlation was observed between baseline JAK2 levels, including JAK2 gene expression, phosphorylated and total JAK2 proteins, and CHR and EMR.

The study concluded that JAK2 levels could serve as an indicator for assessing treatment response to imatinib in individuals with CML, given their involvement in the disease’s pathophysiology.

No funding-related information was available.

Source: https://pubmed.ncbi.nlm.nih.gov/38716474/

Wijaya I, Bashari MH, Reniarti L, et al. (2024). “JAK2 as Predictor of Therapeutic Response in Patients with Chronic Myeloid Leukemia Treated with Imatinib.” Dis Markers. 2024 Apr 12;2024:2906566. doi: 10.1155/2024/2906566. PMID: 38716474; PMCID: PMC11074917.

Imatinib + RIF: Enhanced CP-CML Response

KEY TAKEAWAYS

  • The phase 3 trial aimed to compare arsenic RIF plus imatinib with imatinib alone in patients with newly diagnosed CP-CML.
  • The primary endpoint was MMR.
  • Imatinib plus RIF as initial CP-CML therapy may achieve a deeper molecular response than imatinib alone.

In vitro data confirms the synergistic potential of arsenic compounds combined with imatinib against chronic myeloid leukemia (CML).

Jie Tian and the team aimed to assess the efficacy of arsenic realgar-indigo naturalis formula (RIF) plus imatinib versus imatinib alone in newly diagnosed chronic phase CML (CP-CML) patients through a clinical trial.

This double-blind, multicenter, randomized phase 3 trial involved 191 newly diagnosed CP-CML outpatients. They were randomly allocated to receive either oral RIF plus imatinib (n = 96) or placebo plus imatinib (n = 95).

The primary endpoint was achieving major molecular response (MMR) at 6 months. Secondary endpoints included molecular response 4 (MR4), molecular response 4.5 (MR4.5), progression-free survival (PFS), overall survival (OS), and adverse events (AEs).

The median follow-up lasted 51 months, with recruitment concluding prematurely on May 28, 2020, due to the COVID-19 pandemic. At 6 months and throughout the trial, there was no significant difference in MMR rates between the combination and imatinib arms. Rates of molecular response 4 (MR4) were comparable between both arms.

However, the 12-month cumulative rates of molecular response 4.5 (MR4.5) were 20.8% and 10.5% in the combination and imatinib arms, respectively (P= 0.043).

In the core treatment phase post-2-year analysis, the frequency of MR4.5 was 55.6% in the combination arm and 38.6% in the imatinib arm (P= 0.063). PFS and OS were comparable at 5 years. Safety profiles were similar, with both groups’ rare occurrence of serious AEs.

The analysis showed that combining imatinib with RIF as initial CP-CML therapy might enhance the achievement of a deeper molecular response compared to imatinib alone.

Funding was received by the Yifan Pharmaceutical Co., Ltd.

Source: https://pubmed.ncbi.nlm.nih.gov/38605258/

Clinical Trial: http://www.chinadrugtrials.org.cn/clinicaltrials.searchlistdetail.dhtml

Tian J, Song YP, Zhang GC, et al. (2024) “Oral arsenic plus imatinib versus imatinib solely for newly diagnosed chronic myeloid leukemia: a randomized phase 3 trial with 5-year outcomes.” J Cancer Res Clin Oncol. 2024 Apr 11;150(4):189. doi: 10.1007/s00432-024-05700-x. PMID: 38605258; PMCID: PMC11009770.

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