The latest medical research on Blood Cancer

The research magnet gathers the latest research from around the web, based on your specialty area. Below you will find a sample of some of the most recent articles from reputable medical journals about blood cancer gathered by our medical AI research bot.

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A nomogram model to predict prognosis of patients with hepatoblastoma.

Pediatric Blood and Cancer

Hepatoblastomas (HBs) are malignant liver tumors that most commonly develop in pediatric patients. Microvascular invasion may be a prognosis factor for patients with HBs. This study aimed to construct a model to predict the survival outcome in HBs.

We retrospectively analyzed the clinical data of 311 patients with HBs who underwent surgical resection at our institution between June 2014 and August 2021. First, patients were divided into two groups: those who had pathologic microvascular invasion (n = 146) and those who did not (n = 165). Propensity score-matched (PSM) analysis was carried out between the two groups. The preoperative parameters and overall survival (OS) rate were compared between the two groups. Second, all 311 patients were randomly divided into the training and validation cohort in a ratio of 4:1. A nomogram was created in the training cohort to visualize the prediction of OS. Moreover, the validation cohort was used for validation.

Multivariate analysis suggested that age, histology type, microvascular invasion, multifocality, distant metastasis, and macrovascular involvement are independent prognostic factors for HBs. The nomogram showed good predictive ability in the training and validation cohorts with a C-index of 0.878 (95% CI, 0.831-0.925) and 0.847 (95% CI, 0.757-0.937), respectively. The calibration curve indicated good agreement between the prediction and observation for one-, two-, and three-year OS probabilities.

By combining preoperative imaging results and other clinical data, we established a nomogram to predict OS probability for patients with HB, which could be a potential tool to guide personalized treatment.

Non-neutropenic fever in children with cancer: Management, outcomes and clinical decision rule validation.

Pediatric Blood and Cancer

Fever and infection are an important complication of childhood cancer therapy. Most research and guideline development has focussed on febrile neutropenia, with a paucity directed at non-neutropenic fever (NNF). We describe the clinical presentation, management and outcomes of NNF in children with cancer, and externally validate the Esbenshade Vanderbilt (EsVan) clinical decision rules (CDR) to predict bacteraemia.

Using a prospective database, retrospective data were collected on consecutive NNF episodes (fever ≥38.0°C and absolute neutrophil count >1.0 cells/mm3 ). Sensitivity, specificity and area under the receiver operator characteristic curve (AUC-ROC) of the CDR were compared to derivation study.

There were 203 NNF episodes occurring in 125 patients. Severe sepsis was uncommon (n = 2, 1%) and bacteraemia occurred in 10 (4.9%, 95% confidence interval [CI]: 2.7%-8.8%) episodes. A confirmed or presumed bacterial infection requiring antibiotics occurred in 31 (15%) patients. Total 202 (99%) episodes received at least one dose of intravenous broad-spectrum antibiotic and 141 (70%) episodes were admitted to hospital. Six (3%) episodes required intensive care unit (ICU)-level care and there were no infection-related deaths. The EsVan 1 rule had an AUC-ROC of 0.67, 80% were identified as low risk, and sensitivity and specificity were 50% and 81.5%, respectively, for a risk threshold of 10%.

Serious infection and adverse outcome are uncommon in children with NNF. Many children did not have a bacterial cause of infection identified, but were still treated with broad-spectrum antibiotics and admitted to hospital. National clinical practice guidelines should be developed for this important cohort to enable risk stratification and optimise antibiotic management. Further research is required to determine appropriateness of EsVan CDR in our cohort.

Neurocognitive functioning in children with sickle cell anemia and history of abnormal transcranial doppler ultrasonography.

Pediatric Blood and Cancer

Transcranial doppler (TCD) ultrasonography can be used to identify stroke risk in children with sickle cell anemia. Previous studies have reported mixed findings on neurocognitive outcomes in children with elevated TCD. This study examined associations between TCD velocity and neurocognitive outcomes in children and adolescents without prior history of stroke.

Participants were selected from the Sickle Cell Clinical Research Intervention Program cohort. The highest recorded mean maximum TCD velocity was selected for analysis, along with participant's most recent data from serial neurocognitive surveillance.

A total of 200 children with sickle cell anemia completed neurocognitive testing (109 males, 91 females; mean age 12.7 years [SD = 3.56]). Most participants were prescribed hydroxyurea (72%) at the time of neurocognitive testing and nearly 16% had a history of chronic transfusions prior to neurocognitive evaluation. Mean age at time of highest TCD value was 6.6 years (SD = 2.5) and 13.5% of screenings were abnormal (≥200 cm/s). Mean interval between TCD and most recent neurocognitive evaluation was 6.1 years (±3.5). There were no significant differences in the interval between TCD and neurocognitive testing across normal, conditional, and abnormal groups. Maximum TCD velocity was not significantly associated with neurocognitive outcomes in multivariate models.

History of elevated TCD in the absence of overt stroke should not be considered a risk factor for poor neurocognitive outcomes in children and adolescents with sickle cell anemia on modern disease-modifying therapy.

Mini-hyper CVD + CRIB (condensed rituximab, inotuzumab ozogamicin, and blinatumomab) for refractory pediatric B-acute lymphoblastic leukemia.

Pediatric Blood and Cancer

Relapsed or refractory pediatric patients with B-acute lymphoblastic leukemia (B-ALL) have high rates of toxicities and relapse, and novel therapy ...

Determining drug dose in the era of targeted therapies: playing it (un)safe?

Blood Cancer

Targeted therapies against phosphatidylinositol 3-kinase (PI3K), Bruton's tyrosine kinase (BTK), and B-cell lymphoma-2 (BCL-2) are approved for chr...

Navigating prognostic communication when children with poor-prognosis cancer experience prolonged disease stability.

Pediatric Blood and Cancer

Most pediatric patients and families want clear prognostic information across the illness course. Yet when children with poor-prognosis cancer experience prolonged disease stability, uncertainty can make communication particularly challenging. In this study, we aimed to (i) assess how frequently oncologists communicate about prognosis when high-risk cancer does not progress, and (ii) describe prognostic communication patterns in the context of disease stability.

In this prospective, longitudinal, mixed-methods study, we audio-recorded serial disease re-evaluation conversations between children with poor-prognosis cancer, their families, and their primary oncologists. For this secondary analysis, we conducted content analysis across serial conversations among 16 patient-parent-oncologist triads for whom the patient's disease remained stable over the 24-month study period.

Prognostic communication was absent in >50% of recorded conversations. Overall, it comprised only 4% of dialog time, nearly 90% of which was dialog about prognostic uncertainty; discussion of curability occurred infrequently. Three distinct patterns for prognostic communication emerged: (a) "Don't know" statements, avoiding or deferring prognostication; (b) "Worry" statements, preparing families for possible future disease progression; and (c) relief-caveat statements, celebrating disease stability while balancing positivity with caution.

Oncologists seldom talked about prognosis with high-risk patients during periods of disease stability; yet when they did, they used thoughtful and effective strategies to prepare families for possible future disease progression. Further research is needed to better understand if, how, and when patients and families with stable disease who are high risk for future disease progression prefer to receive information about prognosis.

Diverse mutations and structural variations contribute to Notch signaling deregulation in paediatric T-cell lymphoblastic lymphoma.

Pediatric Blood and Cancer

T-cell lymphoblastic lymphoma (T-LBL) is an aggressive neoplasm closely related to T-cell acute lymphoblastic leukaemia (T-ALL). Despite their similarities, and contrary to T-ALL, studies on paediatric T-LBL are scarce and, therefore, its molecular landscape has not yet been fully elucidated. Thus, the aims of this study were to characterize the genetic and molecular heterogeneity of paediatric T-LBL and to evaluate novel molecular markers differentiating this entity from T-ALL.

Thirty-three paediatric T-LBL patients were analyzed using an integrated approach, including targeted next-generation sequencing, RNA-sequencing transcriptome analysis and copy-number arrays.

Copy number and mutational analyses allowed the detection of recurrent homozygous deletions of 9p/CDKN2A (78%), trisomy 20 (19%) and gains of 17q24-q25 (16%), as well as frequent mutations of NOTCH1 (62%), followed by the BCL11B (23%), WT1 (19%) and FBXW7, PHF6 and RPL10 genes (15%, respectively). This genetic profile did not differ from that described in T-ALL in terms of mutation incidence and global genomic complexity level, but unveiled virtually exclusive 17q25 gains and trisomy 20 in T-LBL. Additionally, we identified novel gene fusions in paediatric T-LBL, including NOTCH1-IKZF2, RNGTT-SNAP91 and DDX3X-MLLT10, the last being the only one previously described in T-ALL. Moreover, clinical correlations highlighted the presence of Notch pathway alterations as a factor related to favourable outcome.

In summary, the genomic landscape of paediatric T-LBL is similar to that observed in T-ALL, and Notch signaling pathway deregulation remains the cornerstone in its pathogenesis, including not only mutations but fusion genes targeting NOTCH1.

Age- and sex-specific rates of gall bladder disease in children with sickle cell disease.

Pediatric Blood and Cancer

Children with sickle cell disease (SCD) have an increased risk for gallstones due to chronic hyperbilirubinemia from hemolysis. Although gallstones are a known complication, there is variability in estimates of disease burden and uncertainty in the association between sex and gall bladder disease (GBD).

This was a retrospective cohort study of children with SCD using administrative claims data (January 1, 2014-December 31, 2018). Population-averaged multivariable panel-data logistic regression models were used to evaluate the association between GBD clinical encounters (outcome) and two exposures (age and sex). Annual GBD risk was calculated using predictive margins, adjusting for disease severity, transfusion frequency, and hydroxyurea exposure.

A total of 13,745 individuals (of 21,487 possible) met inclusion criteria. The population was evenly split across sex (49.5% female) with predominantly Medicaid insurance (69%). A total of 946 individuals (6.9%) had GBD, 432 (3.1%) had a gallstone complication, and 487 (3.5%) underwent cholecystectomy. The annual risk of GBD rose nonlinearly from 1 to 5% between ages 1 and 19 years with no difference between males and females. Cholecystectomy occurred primarily in individuals with GBD (87%), and neither age nor sex was associated with cholecystectomy in this population. High disease severity (compared with low) more than doubled the annual risk of GBD at all ages.

GBD is associated with age but not sex in children with SCD. Neither age nor sex is associated with risk of cholecystectomy. High disease severity increases the rate of GBD at all ages.

Application of a clinical decision rule and laboratory assays in pediatrics: Adult heparin-induced thrombocytopenia.

Pediatric Blood and Cancer

Heparin-induced thrombocytopenia (HIT) is rare among pediatric patients. The diagnosis of HIT depends upon clinical decision tools to assess its pretest probability, supported by laboratory evidence of anti-platelet factor 4 (anti-PF4)/heparin antibodies.

To compare the use of the 4Ts score clinical decision tool, clinical characteristics, and laboratory findings between pediatric and adult patients with suspected HIT.

We compiled all pediatric patients in our center for whom HIT testing was performed during the years 2015-2021. These were compared with a cohort of consecutive adult patients. Laboratory diagnosis of HIT was performed with particle gel immunoassay (PaGIA) as screening test and confirmed by an automated latex-enhanced immunoturbidimetric assay (LIA) and/or by functional flow cytometry assay (FCA).

The cohort included 34 children (under 18 years) and 105 adults. Adults mostly received heparins for thromboembolism prophylaxis and treatment (72.4%, n = 76), and were more frequently treated with low-molecular-weight heparin (LMWH). Children were mostly exposed during cardiopulmonary bypass and extracorporeal membrane oxygenation (ECMO, 61.8%, n = 21), and were more frequently treated with unfractionated heparin (UFH). Compared with adults, children had significantly higher 4Ts scores. Nevertheless, adults had a slightly higher rate of a positive diagnosis of HIT. Six out of 16 adults with confirmed HIT presented with thrombosis (37.5%), whereas all three pediatric patients with HIT presented with thrombosis (p = .087).

4Ts scores are higher in children compared with adult patients for whom laboratory tests for HIT were obtained. A potentially higher incidence of thrombosis in children with HIT may be attributable to the severity of underlying illness.

Myeloma precursor disease (MGUS) among rescue and recovery workers exposed to the World Trade Center disaster.

Blood Cancer

An elevated risk of myeloma precursor disease, monoclonal gammopathy of undetermined significance (MGUS), was identified among Fire Department of t...

CREATE Childhood Cancer Rehabilitation Program development: Increase access through interprofessional collaboration.

Pediatric Blood and Cancer

Cancer and its treatment can lead to functional limitations affecting ongoing development in children and adolescents. We developed a pediatric cancer rehabilitation program that integrates evidence-based rehabilitative care into cancer treatment. The program utilizes the CREATE (collaboration, rehabilitation/research, education, assessment, treatment, evaluation) Childhood Cancer Rehabilitation model. We aim to describe the structural and process components of our rehabilitation program and provide an access and utilization analysis.

To evaluate the rehabilitation program, we identified new patients with oncologic diagnoses from 2002 to 2019 using our database. To evaluate rehabilitative care, descriptive data, including the timing and type of rehabilitation services utilized within 5 years of a child's diagnosis, were collected and reviewed. Statistical analysis focused on change over time.

Among 1974 children assessed, 1580 (80.0%) received care from at least one rehabilitation service. Between 2002 and 2018, the percentage of children receiving rehabilitation services grew significantly throughout all disciplines, except for outpatient speech-language pathology. Utilization differed by age and diagnosis. Integrating therapists in the clinic improved patient access, reduced the time to access outpatient services, and increased the number of attended visits. Additional factors supporting program growth included: identifying leaders, using a prospective surveillance model, education, and program evaluation.

A multimodal interprofessional approach, such as the CREATE model, improves access to and the efficiency of evidence-based rehabilitation services promoting recovery, ongoing development, and quality of life.

Common bone marrow signature in COVID-19-associated multisystem inflammatory syndrome in children: A first-wave small case series experience.

Pediatric Blood and Cancer

The hyper-inflammatory response, also known as multisystem inflammatory syndrome in children (MIS-C), represents a major concern in children with S...