Acta Haematologica最新文献

Introduction: Distinguishing disseminated intravascular coagulation (DIC) from the coagulopathy of liver disease represents a common clinical challenge. Here, we evaluated the utility of two diagnostic tools frequently used to differentiate between these conditions: factor VIII (FVIII) levels and the International Society on Thrombosis and Hemostasis (ISTH) DIC score.

Methods: To this end, we conducted a retrospective chart review of patients with DIC, liver disease, or both. Multiple logistic regression was performed, and receiver operating characteristic curves were generated to calculate the area under curve (AUC) for distinguishing DIC in the setting of liver disease.

Results: Among 123 patients with DIC, liver disease, or liver disease plus DIC, FVIII levels did not differ significantly. ISTH scores were lower in patients with DIC than in liver disease with or without DIC. Addition of several laboratory parameters to the ISTH score, including mean platelet volume, FV, FVIII, international normalized ratio, and activated partial thromboplastin time, improved AUC for distinguishing DIC in liver disease from liver disease alone (AUC = 0.76; p < 0.0001).

Conclusion: We conclude that FVIII levels do not distinguish DIC from liver disease, and ISTH DIC scores are not predictive of DIC in patients with liver disease. Inclusion of additional lab variables within the ISTH DIC score may aid in identifying DIC in patients with liver disease.

Background: Advances in novel therapies have improved outcomes for multiple myeloma (MM) patients and the use of allo-SCT has decreased. Current guidelines no longer support allo-SCT as consolidation therapy for newly diagnosed MM, even in high-risk cases.

Summary: Allo-SCT is now typically considered only within clinical trials for young, high-risk patients with relapsed or refractory MM (RRMM). It has not proven favorable despite its historical use. CAR T-cell therapy and bispecific antibodies have shown promise in treating triple- and penta-exposed/refractory MM, yet relapse remains common with poor survival rates. The efficacy of allo-SCT following BCMA-directed therapy and other new T-cell-directed therapies is unclear. Allo-SCT might be a viable option for eligible patients who relapse after these therapies, or where such options are unavailable. Advancements in reduced-intensity conditioning regimens have led to lower toxicity and transplant-related (TR) morbidity, lower graft-versus-host disease (GvHD), and TR mortality. Expanded use of alternative donors, like haploidentical donors, has yielded comparable outcomes. Better post-transplant GvHD regimens and maintenance strategies to prevent relapse have been developed.

Key messages: This review analyzes available literature to better understand the safety, efficacy, and current role of allo-SCT in managing MM. Newer regimens are needed as routine use of allo-SCT cannot be recommended.

Background: High-dose therapy (HDT) followed by autologous stem cell transplantation (ASCT) has become part of standard of care (SOC) in newly diagnosed multiple myeloma. In this review, we provide a historical perspective on ASCT since its introduction in the 1990s.

Summary: Overall survival (OS) benefit for HDT followed by ASCT was demonstrated in studies comparing HDT with ASCT to standard-dose therapy (SDT) before the era of novel agents. Conditioning is done with melphalan 200 mg/m2. Lower doses (MEL140, MEL150) for older patients with comorbidities are safe and have comparable results. The addition of busulfan to melphalan improves progression-free survival (PFS) but not OS. HDT with ASCT after induction with novel agents prolongs PFS but not OS compared to SDT alone. The benefit is more evident in patients with high-risk cytogenetics. Mobilization can be achieved with granulocyte colony-stimulating factor alone, but is improved with the addition of chemotherapy. Plerixafor reduces mobilization failure and enables sufficient stem cell collection after induction with novel agents. ASCT is safe with a low rate of mortality (1%), and selected patients can be managed as outpatients.

Key messages: HDT followed by ASCT remains part of SOC due to its PFS benefit and relatively low toxicity.

Background: Current guidelines recommend yearly evaluation of glucose metabolism with oral glucose tolerance test (OGTT) in patients with thalassemia major (TM) from their 10th year onwards.

Aims: The aim of the study was to retrospectively evaluate all the OGTT tests performed in a single pediatric center during the last decade and assess the necessity of yearly performed OGTT in patients with TM during the second decade of life.

Results: One hundred and seventy tests performed on 39 patients (24 boys) were included in the analysis. Mean age at the time of each OGTT was 14.15 ± 2.79 years. Seventeen tests (10%) in 8 patients (5 boys) were characterized as impaired glucose tolerance (IGT), whereas in 2 tests (1.18%), fasting glucose values were above 200 mg/dL (diabetes mellitus). Acknowledging the fact that there could be sporadic technical errors, especially in fasting glucose abnormal values, we selectively separated patients with either IGT and abnormal HOMA, MATSUDA, or QUICKI indices or persistently impaired fasting glucose or IGT. With these criteria, 7 patients (4 boys) were identified. No significant differences were observed in demographic and anthropometric parameters, ferritin levels, and mean volume of blood transfused in patients with a definite abnormal glucose metabolism. In order to address selection criteria for performing OGTT without missing a single patient with a definite glucose intolerance, these criteria should include (i) a first OGTT test at the initiation of puberty, (ii) OGTT in any patient with a fasting glucose above 100 mg/dL, and (iii) OGTT in any patient with a ΗΟΜΑ-IR index above 1.85 and yearly onwards. By adopting these criteria, total OGTT tests performed could have been reduced from 170 to only 91, translating to a significant reduction of 46.47%.

Conclusions: Only a small percentage of young patients with transfusion-dependent b-thalassemia exhibit abnormal glucose metabolism during their second decade of life. By adopting specific selection criteria, one may avoid performing this time- and money-consuming test in a significant proportion of patients, albeit without sacrificing timely detection and intervention.

Introduction: Promotion of self-efficacy can enhance engagement with health care and treatment adherence in patients with cancer. We report the outcomes of a pilot trial of a digital health coach intervention in patients with leukemia with the aim of improving self-efficacy.

Methods: Adult patients with newly diagnosed acute myeloid leukemia (AML) and chronic lymphocytic leukemia (CLL) were randomized 1:1 to a digital health coach intervention or standard of care. The primary outcome of self-efficacy was measured by the Cancer Behavior Inventory (CBI) score.

Results: A total of 147 patients (37 AML, 110 CLL) were enrolled from July 2020 to December 2022. In the AML cohort, there was a mean increase in CBI score of 7.03 in the digital health coaching arm compared to a mean decrease of -3.57 in the control arm at 30 days (p = 0.219). There were no significant associations between the intervention and other patient-reported outcomes for patients with CLL.

Conclusion: There were numerical, but not statistically significant increases in self-efficacy metrics in AML patients who received digital health coaching. Although this trial was underpowered due to enrollment limitations during a pandemic, digital health coaching may provide benefit to patients with hematologic malignancy and warrants further investigation.

Introduction: Promotion of self-efficacy can enhance engagement with health care and treatment adherence in patients with cancer. We report the outcomes of a pilot trial of a digital health coach intervention in patients with leukemia with the aim of improving self-efficacy.

Methods: Adult patients with newly diagnosed acute myeloid leukemia (AML) and chronic lymphocytic leukemia (CLL) were randomized 1:1 to a digital health coach intervention or standard of care. The primary outcome of self-efficacy was measured by the Cancer Behavior Inventory (CBI) score.

Results: A total of 147 patients (37 AML, 110 CLL) were enrolled from July 2020 to December 2022. In the AML cohort, there was a mean increase in CBI score of 7.03 in the digital health coaching arm compared to a mean decrease of -3.57 in the control arm at 30 days (p = 0.219). There were no significant associations between the intervention and other patient-reported outcomes for patients with CLL.

Conclusion: There were numerical, but not statistically significant increases in self-efficacy metrics in AML patients who received digital health coaching. Although this trial was underpowered due to enrollment limitations during a pandemic, digital health coaching may provide benefit to patients with hematologic malignancy and warrants further investigation.

Introduction: CMML is a rare neoplasm with overlapping myelodysplastic and myeloproliferative features whose only potential cure is allogeneic hematopoietic cell transplantation (allo-HCT).

Methods: This retrospective study examined 27 CMML patients with high-risk clinical features who underwent first allo-HCT at our institution between 2004 and 2022.

Results: Nineteen patients were diagnosed with the proliferative subtype (CMML-MPN) and 8 with the dysplastic subtype (CMML-MDS). Median OS was 15 months post-HCT (95% CI: 5-71); OS at 1, 3, and 5 years was 52%, 35%, and 35%, respectively. Compared to those with CMML-MPN, patients with CMML-MDS had longer OS (median, 8.6 vs. 0.9 years; p = 0.025), RFS (4.4 vs. 0.5 years; p = 0.021), and GVHD-free, relapse-free survival (GRFS, 9.4 vs. 3.4 months; p = 0.033) as well as lower 1-year NRM (13 vs. 47%; p = 0.043), with the statistical significance of this CMML subtype effect maintained in multivariable models. High-risk cytogenetics were associated with shorter GRFS in the univariable (median, 3.1 vs. 6.2 months; p = 0.013) and multivariable (HR = 4.88; p = 0.006) settings.

Conclusions: Patients who underwent transplant for CMML-MDS experienced substantially better outcomes than those transplanted for CMML-MPN. Future studies are needed for transplantation optimization in CMML, especially CMML-MPN.

Background: Atrial fibrillation (AF) is a prevalent cardiac arrhythmia associated with a significantly increased risk of systemic thromboembolism and stroke. Anticoagulation therapy, particularly with direct oral anticoagulants, has become the standard for stroke prevention but comes at the cost of an increased bleeding risk. With the introduction of effective alternatives to anticoagulation, such as percutaneous left atrial appendage occlusion, bleeding risk stratification has become essential to guide therapeutic decision-making. Conventional statistical methods have been used for bleeding risk stratification scores, such as HEMORR2HAGES, HAS-BLED, and ATRIA. However, these methods may inadequately address the multifactorial nature of bleeding risk in diverse patient populations, and their overall performance has been suboptimal. Summary and Key Messages: Recent advancements in machine learning (ML) offer promising opportunities to enhance bleeding risk prediction and optimize anticoagulation therapy. This review explores ML applications in AF patients receiving anticoagulation therapy, focusing on the development and validation of ML-based bleeding risk scores. These models have demonstrated improved predictive performance compared to traditional tools, leveraging complex datasets to identify nuanced patterns and interactions. Furthermore, ML-driven tools in warfarin management, including dose prediction, optimization of time in the therapeutic range, and the identification of drug-drug interactions, show significant potential to enhance patient safety and treatment efficacy.

Background: Venous thromboembolism (VTE), including deep vein thrombosis and pulmonary embolism, remains a leading cause of cardiovascular morbidity and mortality. Artificial intelligence (AI) holds promise for potential improvement of risk stratification, diagnosis, and management of VTE. Summary: This narrative review explores the applications, benefits, and limitations of AI in VTE management. AI models were shown to outperform conventional methods in identifying high-risk candidates for VTE prophylaxis treatments in several postsurgical settings. It has also been demonstrated to be efficient in the early detection of VTE events, particularly through point-of-care AI-guided sonography and computer tomography image processing. Data biases, model transparency, and the need for regulatory frameworks remain significant limitations in the full integration of AI into clinical practice. Key Messages: AI has the potential to improve VTE care by enhancing risk stratification and diagnosis. The integration of AI-driven models into clinical workflows has the potential to reduce costs, streamline diagnostic processes, and ensure effective management of VTE. Safe and effective integration of AI into VTE care requires addressing its limitations, such as interpretability, privacy, and algorithmic bias.