Hematological Research Articles

Cancer Stage Geospatial Analysis to Optimize Educational Activities in Pediatric Oncology and Hematology: A Retrospective Cohort Study.

Enhancing therapy outcomes in pediatric oncology and hematology relies on robust epidemiological surveillance. This study aimed to estimate cancer stage-related survival in pediatric patients with lymphoma and solid tumors by assessing changes over time and identifying spatial clustering of patients diagnosed at advanced stages. This retrospective observational cohort study included pediatric cancer patients treated in a single Greater Poland center, constituting 9% of Polish children from 2004 to 2017. The incidence, cancer stage, and patient coordinates were analyzed. The follow-up period ranged from 5 to 18years. Survival differences across tumor stages were evaluated using Kaplan‒Meier curves, log-rank tests, and trend analysis. Geographical analysis was performed with Kulldorff's scan statistics and the Bernoulli model. Among 1094 diagnosed patients, 511 with lymphoma and solid tumors were eligible. There was a decreasing trend in advanced-stage diagnoses (p = 0.0001), with a nearly twofold increase in low-stage diagnoses (OR = 1.98 [1.22; 3.24], p = 0.0061) from 2009-2011 to 2015-2017. Hazard ratios for neuroblastoma and sarcoma patients were more than fourfold greater, while survival differences were not significant for patients with nephroblastoma, germ cell tumors, or lymphoma. This study shows cancer stage-related survival dynamics. A substantial decrease in advanced-stage diagnoses over time emphasizes improved early detection. Geographical analysis pinpointed clusters with prevalent late-stage diagnoses, offering a practical tool for targeted educational interventions. The study underscores the pivotal impact of cancer stage on survival outcomes, emphasizing the need for ongoing surveillance and tailored interventions to further optimize pediatric oncology and hematology care.

Use of non-specific immunoglobulins in Catalonia in three third-level hospitals: a descriptive analysis of a hospital-prescribed medication registry

BackgroundThe increasing use of non-specific immunoglobulins (NSIGs) and their current shortage show a need for NSIGs’ use prioritization. Data from a clinical perspective are necessary, mainly for pediatric patients.ObjectivesThe aim of the study was to assess the level of clinical evidence (LoE) of the indications that NSIGs are used for, the reasons for discontinuation, and the costs invested.MethodsA retrospective multicentric study was conducted on NSIG incident users between September 2019 and December 2021 retrieved from the Registry of Patients and Treatments (RPT) from Catalonia (Spain). LoE was categorized as A) authorized indications, B) unauthorized with scientific support, C) unauthorized without support, and D) unknown (UNK), following local and the United Kingdom’s guidelines as a sensitivity analysis. We also estimated overall spending and costs per patient visit.ResultsA total of 400 patients were included (17.3% pediatric), with a mean follow-up of 122.1/person-years for adults. The most frequent indications were nervous system and blood diseases. Almost all pediatric patients (56; 81.2%) were treated under A-level indications, as for 217 (65.6%) adults. In the sensitivity analysis, the A-level usage rate decreased to one-third and the B-level usage rate increased by 2–3 times. Furthermore, 37.8% (151) of individuals discontinued. This was predominantly due to remission or no response. The total costs were 868,462.6€/year, with median spending per visit amounting to 1,500€ for adults and 700€ for pediatric patients.ConclusionNSIGs are used in clinical practice mainly for approved indications; however, non-approved indications are still an important issue. This could represent a significant economic burden on the healthcare system, focusing on the pediatric population and those at risk for discontinuation with alternative therapeutic options.

Особенности системы гемостаза при истинной полицитемии у детей

Patients with polycythemia vera (PV) are at an increased risk for thrombosis. Abnormalities in coagulation parameters and global hemostasis tests can help with hypercoagulation recognition in PV. The purpose of this research was to analyze the features of changes in laboratory parameters of the hemostasis system in pediatric patients with PV. Materials and methods used: a single-center retrospective cohort study of clinical records of 32 (11 (35%) girls/21 (65%) boys aged 11.5 [0.8; 17.5] y/o) pediatric (all under the age of 18 y/o) PV patients who were admitted at the National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev (Moscow, Russia) in Jan. 2012-June 2024 was conducted. Laboratory parameters were determined prior to the beginning of the antiplatelet and cytoreductive therapy. Results: in 20 (62.5%) and 4 (12.5%) patients, mutations in the JAK2V617F and JAK2 ex 12 genes were detected, respectively, the remaining 8 (25%) children were JAK2-negative. In total, there were 8 (72%) JAK2-positive girls and 15 (71%) boys; gender did not statistically significantly affect the presence of a driver mutation (p=0.939). Both the median and the mean values of the main parameters of the blood coagulation system were within the reference values. The thromboelastography parameters did not deviate from the reference values for the parameters. According to the results of the thrombodynamics study, the average value of the initial clot growth rate among all patients was increased and amounted to 58.3±5.07 μm/min, the steady-state velocity also increased among all patients to 29.5±4.3 μm/min, the median and the mean values of the remaining parameters were within the reference intervals. Conclusion: the parameters of standard coagulogram and thromboelastography tests in pediatric PV patients had no deviations. The average values of thrombodynamics (initial and steady-state clot growth rate) were increased, which potentially indicates the hypercoagulation.

Технические аспекты лапароскопического удаления нейрогенных опухолей абдоминальной локализации у детей

Progress in a modern pediatric oncological surgery, including neuroblastoma surgery, is characterized by increased role of the minimally-invasive approach. Authors represent standardized technical aspects of abdominal neuroblastoma laparoscopic resection. The purpose of this research was to systematize the technical aspects of laparoscopic resection for pediatric abdominal neuroblastoma, also in cases with Image-Defined Risk Factors (IDRF). Methods used: pre- and intraoperative data of 181 patients (85 (47%) males/96 (53%) females) of the median age of 14 [5-51] (1-209) months old with abdominal neuroblastoma who had received laparoscopic tumor resection at the National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev (Moscow, Russia) in Jan. 2018-Jan. 2024 were analyzed. Technical features of laparoscopic surgery procedure, also in cases with IDRF have been systematized as follows: patient position on the operating table, trocars placement, visualization of surgical field, dissection plan. Results: maximal tumor length was 3,6 [2,6-5] (0,7-12,6) cm, maximal tumor volume was 16,8 [4-40] (0,5-557) ml. IDRF were identified in 53 (29%) cases, wherein two and more IDRFs were in 19 (10%) patients. The surgical intervention duration was 100 [70-140] (35-725) minutes, intraoperative blood loss was 5 [5-18] (5-3600) ml, intraoperative transfusion was 0 [0-0] (0-890) ml. Intraoperative complications were observed in 6 (3%) patients, conversion was required in 7 (4%) cases. Complete tumor resection (98% to 100%) was achieved in 163 (90%) cases. Conclusion: the accumulated experience of minimally invasive surgery for neuroblastoma indicates the demand for reviewing of the current indications for these surgical procedures as well as a precise investigation of the effect of each IDRF on the difficulty and risk of surgery. In this Article Authors have described standardized technical features of abdominal neuroblastoma laparoscopic resection. These technical features are used in prospective clinical trial whilst the purpose of this research was to design the difficulty scoring system for such surgery (ClinicalTrials.gov ID NCT06296732).

Systematic inflammatory indicators and clinical management of exogenous endophthalmitis due to metal penetrating injury of eyeball

ObjectiveTo evaluate systemic inflammatory markers in cases of exogenous endophthalmitis caused by metal foreign bodies after penetrating eye injury and identify risk factors for poor control post-initial emergency surgery.MethodsTwenty-nine patients with exogenous endophthalmitis underwent emergency surgery with vitrectomy at Xiamen Eye Center (2016–2024). Evaluations included systemic inflammatory markers, microbiology, clinical presentation, treatment strategies, complications, prognostic factors, and visual outcomes. Correlation analysis was performed between blood test results and disease control after initial surgery.ResultsThe median patient age was 44.8 ± 16.9 years, with an average treatment delay of 6.1 ± 10.6 days. Males represented 86.2% (n = 25), and 41.3% (n = 12) had metallic intraocular foreign bodies, which correlated with faster symptom onset (p < 0.05) and higher inflammatory markers (WBC, neutrophils, basophils, CRP; p < 0.05). Primary lens extraction and intraoperative vancomycin did not significantly improve control (p > 0.05). Poor control post-vitrectomy was positively correlated with intraocular foreign bodies (R = 0.39, p < 0.05) and negatively with lymphocyte and monocyte counts (R = −0.43, p < 0.05; R = −0.46, p < 0.05). Early intervention within 2 h of symptom exacerbation reduced complication risk (AUC = 0.708, 95% CI: 0.547–0.838, p = 0.047).ConclusionMetallic intraocular foreign bodies provoke stronger systemic inflammatory responses, posing control challenges, especially in patients with lower immune resilience. Timely vitrectomy during initial emergency surgery is crucial for managing endophthalmitis.

A Pilot Study in a Recently Established Childhood Cancer Center for Evaluating the Priority of National Pediatric Cancer Registry

Background: Pediatric cancers account for nearly 2% of all cancers worldwide. In 2018, there were approximately 200,000 newly diagnosed cases of childhood cancer. Objectives: The aim of this study was to assess the need for a national pediatric cancer registry based on case incidence. Methods: This pilot study was conducted on 100 children with cancer who were treated in a recently established pediatric hematology oncology department of Golestan Hospital. Demographic and epidemiological data were collected and analyzed using SPSS software version 25. Results: Sixty-four percent of children with cancer were girls, and the average age of the patients was 7.6 ± 0.42 years. The most common types of cancer diagnosed were leukemia, brain tumor, lymphoma, and retinoblastoma respectively. Metastasis was confirmed in 11 cases. The most prevalent clinical manifestations of cancer included fever, weakness, loss of appetite, paleness, and weight loss. Conclusions: The results of this study align with global rates and data. Based on the findings, the authors emphasize the necessity and priority of establishing a pediatric cancer registry in Iran's health ministry. Therefore, all healthcare workers and specialists in pediatric hematology and oncology should make concerted efforts to establish this registry.

Taste alteration, Food Habits and Nutritional Status of Childhood Cancer Survivors

Aim: This study was carried out to examine the taste alteration, food habits, and nutritional status of childhood cancer survivors. Materials and Methods: The research is descriptive and cross-sectional. The study sample was formed by children between the ages of 8 and 18 who ended cancer treatment between March 2021 and 2022, followed by the pediatric hematology and oncology outpatient clinic of a University Medical Faculty Oncology Hospital. Child Introduction Form, Taste Alteration Scale for Children with Cancer Receiving Chemotherapy (TAS-CRC), Mediterranean Diet Quality Index (MDQI), and Subjective Total Taste Acuity Scale (STTA) were used to collect data. Results: It was determined that 80.4% of children had a Body Mass Index (BMI) score between -2 and +2 (normal), 9.8% were above ≥+2 (obese) and 7.6% were under ≤-2 (malnutrition). Analysis showed that 18.5% of the children had a very low nutrition quality (≤3), while nutrition quality was moderate in 48.9% (4-7) and good in 32.6% (≥8). Conclusion: It was found that there was no statistically significant difference between the weight Z score of the children, the current height Z score and the BMI Z score, and the average score TAS-CRC.

Fecal occult blood is associated with an increased risk of cerebral small vessel disease in elderly patients

Background and purposeStudies have shown that fecal occult blood is associated with an increased risk of ischemic stroke, but the relationship between fecal occult blood and cerebral small vessel disease (CSVD) remains largely unknown. This study aimed to identify predictors for CSVD, with fecal occult blood and its proxies as potential influencing factors in elderly patients. MethodPatients aged 65 years or older with various chronic diseases were enrolled. The presence of CSVD was evaluated by brain MRI results. Fecal occult blood was measured by fecal immunochemical test. Logistic regression analysis was used for the association between the presence of fecal occult blood and the risk of CSVD. ResultsLogistic regression analysis indicated that a prevalence of positive fecal occult blood was related to CSVD (Model 1, adjusted OR=1.63, 95% CI: 1.15–2.29, P=0.006). We subsequently grouped all subjects as positive fecal occult blood (259, 16.48%) and negative fecal occult blood (1313, 83.52%), and logistic regression analysis indicated that a prevalence of CSVD was related to positive fecal occult blood (Model 2, adjusted OR=1.50, 95% CI: 1.08–2.08, P=0.015). In addition, the ratios of lacunes (67.18% vs. 53.85%, P<0.001) and enlarged perivascular spaces (43.63% vs. 34.42%, P=0.005) were higher in patients with positive fecal occult blood than in controls. ConclusionsThe presence of fecal occult blood is probably related to the risk of CSVD in elderly patients and could be used as a screening tool for CSVD in elderly populations.

Is Allogeneic Transplantation for Sickle Cell Disease Still Relevant in the Era of Gene Therapy?

Sickle cell disease (SCD) is the most common inherited blood disease. Disease-modifying therapy and supportive care have improved the survival of children with SCD in the United States and Europe. Yet, adults with SCD continue to have high risks of morbidity and early death. Recently, two FDA-approved genetic therapies offer the potential for a short-term decrease in acute vaso-occlusive pain events if not cure. Allogeneic hematopoietic cell transplantation (alloHCT) is also curative, but until recently was constrained by limited donor availability and the risks of graft-versus-host disease, graft rejection, and death. Importantly, recent advances have attenuated these barriers. Here, we discuss the current state of therapies with curative intent for SCD. Both genetic therapy and alloHCT offer the potential for cure for most with SCD. However, the cost (about five times higher), the current need for myeloablation, and associated late-health effects may make genetic therapies less favorable choices than alloHCT.

Impact of a two step antimicrobial stewardship program in a paediatric haematology and oncology unit

Objective: To describe the implementation of a multi-step antimicrobial stewardship program in a haemato-oncology and stem cell transplantation program unit. Methods: Pre-post quasi-experimental study with two interrupted time-series analyses, conducted between 01/01/2019 and 31/12/2022 in the Paediatric Haemato-Oncology Unit of the Padua Paediatric Hospital. The interventions were: (1) 02/2020: dissemination of febrile neutropenia clinical pathways, (2) April 2021: provision of the clinical pathways via a customized App (Firstline.org) and implementation of a twice-a-week prospective audit and feedback. The main outcome was antibiotic consumption measured by days of administered therapy (DOTs)/1000 patients’ days for all antibiotics and most used molecules. Results: The first intervention (clinical pathways) resulted in a decrease in the overall antibiotic use by the haemato-oncology unit, with an abrupt reduction of 3-gen cephalosporins in favor of piperacillin-tazobactam, as indicated by the clinical pathways. Meropenem and glycopeptide use did not vary. The second intervention (antimicrobial stewardship) further decreased total antibiotic consumption, and a significant decline in meropenem, amikacin, and glycopeptides was achieved. Conclusions: Multi-step stewardship based on guidelines dissemination, multidisciplinary team intervention and collaboration (“handshake” stewardship) was highly effective in optimizing guidelines adherence and reducing overprescriptions in a fragile patient cohort.

Serum Vitamin D in Children with Hemophilia A and Its Association with Joint Health and Quality of Life

Background/Objectives: Hemophilia A is an X-linked recessive illness produced by a deficiency of coagulation factor VIII. This study aimed to evaluate serum vitamin D in hemophilic pediatric patients and its correlation with joint health and quality of life. Methods: This case-control study was performed on ninety children under the age of 18 years old and separated into two groups: study group of 45 children with hemophilia A and control group of 45 healthy children at an outpatient pediatric hematology clinic at the Beni-Suef University hospitals. Results: Serum vitamin D levels were significantly lower in hemophilia A patients than in controls (p &lt; 0.001). The level of serum vitamin D was deficient in 38 (84.4%), insufficient in 4 (8.8%) and sufficient in 3 (6.6%) in the study group while deficient in 8 (17.7%), insufficient in 16 (35.5%) and sufficient in 21 (46.6%) in the control group. Total hemophilia joint health score (HJHS) had a significant negative correlation with serum total calcium (R = −0.31, p = 0.038) and serum vitamin D level (R = −0.974, p &lt; 0.001) while also positively correlated with alkaline phosphatase (R = 0.834, p &lt; 0.001). A quality-of-life index that is specific to total hemophilia (Haemo-Qol/Haem-A-QoL) had a significant positive correlation with total hemophilia joint health score (HJHS) (R = 0.934, p &lt; 0.001) and negatively correlated with serum vitamin D level (R = −0.924, p-value lower than 0.001), alkaline phosphatase (R = 0.842, p &lt; 0.001), and severity of hemophilia (R = 0.67, p &lt; 0.001). Conclusions: patients with hemophilia A had lower vitamin D levels than healthy controls. The severity of vitamin D deficiency is related positively to (HJHS) hemophilia and quality of life hemophilia cases according to Haemo-QoL.

Risk factors of asparaginase-associated pancreatitis in children with acute lymphoblastic leukaemia

Background: Acute lymphoblastic leukaemia (ALL) is the top childhood cancer, with survival rates of 80–85%. Attention now focuses on treatment complications. Pancreatitis, linked to Asparaginase in ALL therapy, remains poorly understood despite its recognition. The main objective of the study was to identify the risk factors of pancreatitis in children with acute lymphoblastic leukemia receiving L-Asparaginase during induction chemotherapy. Methods: A prospective observational study was conducted at BSMMU's Paediatric Haematology and Oncology Department from March 2018 to February 2019. Newly diagnosed ALL cases in children aged 1 to 17.9 years were included, excluding those &lt;1 or &gt;18 years, with prior pancreatitis, or relapsed cases. Informed consent was obtained from parents/legal guardians. Diagnosis relied on clinical, CBC, bone marrow and immunophenotyping. Induction chemotherapy followed the UK ALL 2003 protocol for 35 days, with regular follow-ups monitoring abdominal symptoms and laboratory markers. USG of the abdomen was performed if pancreatitis symptoms or elevated serum amylase/lipase occurred post-L-asparaginase administration. Results: Among 80 patients, pancreatitis affected 3 (3.8%) after the 2nd, 3rd and 7th doses of L-asparaginase, independent of individual or cumulative dosing, induction phase or concomitant medications. Age, sex, initial WBC count, ALL lineage, treatment regimen and CNS status were not statistically linked to pancreatitis incidence. Clinical manifestations included abdominal pain, tenderness, nausea, vomiting and fever, alongside elevated serum amylase and lipase levels, supported by consistent ultrasonographic findings. Despite these symptoms, no pancreatitis-related mortalities were observed, however, overall mortality during induction therapy reached 20%, unrelated to pancreatic complications. Conclusions: In this study no significant risk factor could be identified for developing pancreatitis in ALL patients treated with asparaginase. Further studies with large sample size are required to predict who will develop pancreatic toxicity from asparaginase.

Revue des atteintes dermatologiques atypiques et/ou systémiques en lien avec les inhibiteurs de checkpoints immunologiques

Immunological checkpoint inhibitors are now part of the oncological therapeutic arsenal in many solid cancers and malignant blood diseases, at the cost of immuno-mediated toxicities, of which dermatological disorders are among the most frequent. The most common, following treatment with anti-PD1 or anti-CTLA4, are maculopapular erythema, pruritus, vitiligo, or lichenoid lesions, but other more atypical conditions may lead to the internist being called upon. Here, we present a case series of these less common dermatological manifestations including fasciitis, dermatomyositis, scleroderma, granulomatosis and immune-induced vasculitis. Some manifestations appear similar to the primary forms or seem to correspond to paraneoplastic syndromes, but some diagnostic and therapeutic particularities are specific to ICI toxicity that the internist must be aware of.

Pharmacovigilance study of famciclovir in the Food and Drug administration adverse event reporting system database

Famciclovir, mainly used to treat herpes zoster, is rapidly transforms into penciclovir when administered orally. Our study evaluated adverse events (AEs) associated with famciclovir by mining data from the publicly available Food and Drug Administration Adverse Event Reporting System (FAERS) database, providing a reference for clinical safety. Disproportionality analysis (including reported odds ratio and proportional reporting ratio) and Bayesian methods (including Bayesian Confidence Propagation Neural Network and Gamma-Poisson Shrinkage) were used to quantify the AE signals associated with famciclovir. A total of 17,652,186 case reports were obtained from the FAERS database, and 432 famiclovir-related AEs were identified. Nausea, headache, altered mental status, vomiting, and dizziness were found to be the most common Aes, corresponding to those reported in the Food and Drug Administration (FDA) drug labelling and clinical trials. Our study found some potential AEs of famciclovir that were not mentioned in the FDA drug labelling, such as toxic encephalopathy, encephalopathy, ataxia, dysarthria, dementia, cerebral infarction, tremor, purpura, skin ulcers, acute pancreatitis, rhabdomyolysis, muscle twitching, increased blood urea, lowered blood pressure, hepatitis, disease recurrence, drug interactions, and pancytopenia. Our study identified potential famciclovir AE signals, providing insights for physicians to reduce possible side effects and promote the safe implementation of the drug in clinical settings.

A Pilot Study of Intelligent Control of Nanorobots using AI &amp; ML

Recent advancements in artificial intelligence (AI) and nanotechnology have enabled the development of intelligent nanorobots, offering a transformative approach to disease management, particularly in the fields of neuro pharmaceutics and blood disease monitoring. This Study explores the potential of nanoparticles in biomedical engineering, specifically focusing on their application as nanorobots capable of crossing the blood-brain barrier (BBB) for targeted payload delivery in brain tumors and neurological disorders such as Alzheimer's and Parkinson's diseases. These nanorobots, designed with sensors, actuators, power sources, and communication systems, utilize AI algorithms for precise navigation within the bloodstream. Materials like graphene and gold nanoparticles ensure biocompatibility, enhancing both safety and efficacy in delivering targeted therapies. Furthermore, AI-driven nanorobots are revolutionizing cancer drug delivery, reducing toxicity, and improving therapeutic outcomes. Additionally, they show promise in cardiovascular health monitoring for early disease detection. However, challenges such as regulatory and technological barriers persist, necessitating ongoing research to realize the full potential of AI-powered nanorobots in personalized and precision medicine.

Ifosfamide-Induced Encephalopathy Managed Successfully with Methylene Blue in a Child with Wilms Tumor: A Case Report and Review of the Literature

Introduction: Ifosfamide is a crucial element in various chemotherapy protocols for the treatment of diverse solid tumors in both children and adults. Despite its efficacy, ifosfamide is associated with a rare yet severe adverse effect known as ifosfamide-induced encephalopathy (IIE), with a spectrum of signs and symptoms spanning from mild lethargy to coma or death. While approximately 13% of pediatric patients undergoing ifosfamide treatment experience IIE, consensus regarding its management remains controversial. Case Presentation: We present the case of a 4-year-old girl with stage 4 Wilms tumor and lung metastases who underwent radical nephrectomy followed by UH-1 chemotherapy. Imaging post-chemotherapy initially showed tumor clearance but later revealed relapse and lung metastasis. Following the initiation of the adjusted ICE protocol (ifosfamide, etoposide), the patient developed IIE, which was successfully treated with methylene blue. Conclusions: We strongly advise pediatric oncologists and hematologists to proactively consider the potential occurrence of IIE. Before starting ifosfamide therapy, patients should be assessed for possible risk factors associated with IIE. Considering methylene blue as a prophylactic measure is recommended for patients with significant risk factors. In cases where IIE manifests, using Methylene Blue to alleviate and reverse its signs and symptoms should be considered. Moreover, we highly encourage further research endeavors to validate methylene blue's effectiveness and determine its optimal dosage range, especially concerning pediatric patients.

One of the Shining Stars of Ankara University Faculty of Medicine, the Founder of Pediatric Hematology and Oncology in Türkiye: Prof. Dr. Ayhan (Okçuoğlu) Çavdar

One of the Shining Stars of Ankara University Faculty of Medicine, the Founder of Pediatric Hematology and Oncology in Türkiye: Prof. Dr. Ayhan (Okçuoğlu) Çavdar

Microalbuminuria in Children With Sickle Cell Disease in the Eastern Province of Saudi Arabia.

Sickle cell disease (SCD) complications, such as sickle cell nephropathy (SCN), may begin in childhood and progress insidiously to chronic kidney disease in adulthood. In Saudi Arabia (SA), there is a lack of studies evaluating kidney function in children with SCD. This study aims to assess microalbuminuria (MA) as an early marker of renal dysfunction in SCD children living in the Eastern region of SA, to potentially institute appropriate early treatment. A prospective cross-sectional study was conducted on 114 Saudi children with SCD under the age of 14 years who attended the pediatric hematology clinic for routine follow-up. Demographic and clinical information were collected from the patients and their parents, who provided informed consent. Morning urine samples were collected and tested for the presence of MA using the urinary albumin/creatinine ratio (ACR). Blood samples were also collected for basic laboratory investigations. The prevalence of MA and its correlation with various clinical and laboratory data were analyzed. Additionally, a comparison of clinical characteristics and MA was conducted between children originating from the Southwestern (SW) and Eastern regions of the country, all of whom lived in the Eastern Province. A total of 114 children with SCD were included in the study. The mean age was 8.8 ± 3.2 years, with a male-to-female ratio of 1.3:1. Based on their region of origin, they were divided into two groups: Eastern (n = 26/114) and SW (n = 88/114). MA was detected in 28 patients (24.6%), with no significant difference in prevalence between the two groups. There was no significant statistical difference in clinical and laboratory data between the groups, except for hemoglobin F (HBF) levels and the use of hydroxyurea (HU). HBF levels were significantly higher in children from the Eastern region, while more SW patients used HU. No correlation was found between MA and any of the studied variables. MA is common in children with SCD in the Eastern region of SA, with no difference in its prevalence between children of the two different ancestries carrying the Arab-Indian (AI) and African haplotypes. It is not associated with any of the studied clinical variables in this report. Further studies are needed to confirm these findings.

Minimal residual disease detection for acute lymphoblastic leukaemia in peripheral blood-Are we there yet?

Can peripheral blood be used to detect residual disease in acute lymphoblastic leukaemia (ALL) when we increase the sensitivity of the method used? Bendig etal. found that a larger amount of material and the use of next-generation sequencing (NGS) detects MRD in peripheral blood in up to half of patients with B-cell precursor ALL (BCP-ALL) where routine examination was negative. However, a negative result does not exclude the presence of residual disease and thus still limits the use of peripheral blood. Commentary on: Bendig etal. Next-generation sequencing and high DNA input identify previously missed measurable residual disease in peripheral blood of B-cell precursor acute lymphoblastic leukaemia. Br J Haematol 2024 (Online ahead of print). doi: 10.1111/bjh.19834.

Prediction model for bone erosion in rheumatoid arthritis based on musculoskeletal ultrasound and clinical risk factors.

Progressive bone erosion (BE) is a prominent manifestation of structural damage in rheumatoid arthritis (RA). This study aimed to construct and validate a BE prediction model (BEPM) for RA based on musculoskeletal ultrasound and clinical risk factors. A total of 312 RA patients without BE were consecutively collected and followed up for 2years, who were divided into BE group and non-bone erosion group, as confirmed by two radiology experts based on at least two imaging techniques. Relevant clinical information, such as anti-cyclic citrullinated peptide (ACCP) antibody and disease duration, was collected. Musculoskeletal ultrasound examinations were performed on all patients upon admission. Univariate and binary logistic regression analyses were conducted to identify risk factors for BE and to establish the prediction model. The calibration, diagnostic efficacy, and clinical effectiveness of BEPM were evaluated by calibration curve plots, receiver-operating characteristic curve, and decision curve analysis (DCA). Binary logistic regression analysis screened four variables, including synovial hyperplasia, synovial blood flow, ACCP + , and disease duration, into the prediction model. BEPM exhibited good diagnostic performance in both the training and validation groups, with area under the curve values of 0.949 (95% CI 0.924-0.973) and 0.965 (95% CI 0.935-0.996), respectively. Using 0.376 as the optical cutoff value for BE, the sensitivity, specificity, and Youden index of the model in the training group were 86.0%, 90.4%, and 76.4%, respectively. In the DCA curves, BEPM indicated overall good population benefit. BEPM demonstrates high diagnostic performance and clinical utility, and may be a useful clinical model for predicting BE in patients with RA. Key Points • Synovial hyperplasia, synovial blood flow, ACCP+ , and disease duration are independent risk factors for bone erosion in RA patients. • BEPM exhibits good diagnostic performance in both the training and validation groups. • Combining musculoskeletal ultrasound parameters with key clinical risk factors to construct a bone erosion model is feasible.