Achondroplasia (ACH) is a common inherited skeletal dysplasia (inherited dwarfism) that compromises quality of life across the lifespan. In 2021, vosoritide became the first approved precision therapy for ACH and is now available in more than 40 countries. Compared with prior symptomatic measures, vosoritide has demonstrated favorable efficacy and a reassuring safety profile. Nevertheless, existing international ACH guidelines largely emphasize complication management and symptomatic care, and there is no unified consensus on pharmacologic therapy. To address this gap, an international expert group developed the International Consensus Guidelines for the Implementation and Monitoring of Vosoritide Therapy in Patients with Achondroplasia providing systematic recommendations that span the continuum of care - from initial patient contact and pre-treatment assessment to medication counseling, injection training, and long-term outcome monitoring. These recommendations complement and refine current management and nursing protocols for individuals with ACH and offer practical guidance for clinicians across diverse regions. This article highlights key elements of the guideline to provide evidence-based support and clinical direction for healthcare professionals in China treating children with ACH using vosoritide.
Humans ; Achondroplasia/drug therapy* ; Consensus ; Practice Guidelines as Topic ; Child

OBJECTIVE: To analyze the clinical characteristics, treatment, and prognosis of seven pediatric patients with Acute myeloid leukemia (AML) positive for the t(16;21)(p11;q22) FUS::ERG fusion gene. METHODS: A retrospective analysis was carried out on the clinical data, treatment, and prognosis of seven AML patients with t(16;21)(p11;q22) FUS::ERG fusion gene admitted to Henan Children's Hospital between June 2015 and November 2024. Relevant literature was also reviewed. This study was approved by the Medical Ethics Committee of the Hospital (Ethics No.: 2024-102-001). RESULTS: Among 297 pediatric patients with AML, 7 cases (2.36%) were positive for the t(16;21)(p11;q22) FUS::ERG fusion gene, including 3 males and 4 females, with a median age of 11 years (range: 3 ~ 12 years). According to the FAB classification, these included 1 case of M2, 3 cases of M5, and 3 cases of AML-not otherwise specified (non-M3). All 7 patients were found to harbor the t(16;21)(p11;q22) translocation, with 3 cases showing additional chromosomal abnormalities. Immunophenotyping revealed universal expression of CD13, CD33, CD34, and CD117, with partial expression of CD56, CD4, CD64, CD123, CD15, CD38, CD11b, HLA-DR, cMPO, and CD16. One patient achieved complete remission (CR) after the first course of DAE (cytarabine + daunorubicin + etoposide) induction chemotherapy but relapsed and discontinued the treatment. Six patients received DAH (cytarabine + daunorubicin + homoharringtonine) induction therapy, of whom 2 achieved CR after two courses and underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT), resulting in an overall CR rate of 42.86%. Five children did not receive allo-HSCT and had a median overall survival of 9 months (range: 6 ~ 18 months). Two children who underwent transplantation achieved bone marrow morphological and molecular biological relapse at 6 and 9 months post-transplantation, respectively. After receiving combined chemotherapy and donor lymphocyte infusion, one child failed to achieve remission and died at 22 months post-transplantation, while the other has been followed up to date with positive fusion gene status. Their overall survival was 25 months and 30 months, respectively. CONCLUSION The t(16;21)(p11;q22) FUS::ERG fusion gene is rare in pediatric AML and associated with poor prognosis. Allo-HSCT may mitigate the adverse prognostic impact of the FUS::ERG fusion gene and contribute to prolonged survival.
Humans ; Male ; Child ; Female ; Leukemia, Myeloid, Acute/drug therapy* ; Oncogene Proteins, Fusion/genetics* ; Translocation, Genetic ; Retrospective Studies ; RNA-Binding Protein FUS/genetics* ; Chromosomes, Human, Pair 16/genetics* ; Adolescent ; Child, Preschool ; Chromosomes, Human, Pair 21/genetics* ; Prognosis ; Treatment Outcome

Abstract Background: Optimal mucosal visibility during esophagogastroduodenoscopy (EGD) is critical for diagnostic accuracy but is often impaired by the presence of mucus and bubbles. This study aimed to compare the efficacy and safety of four premedication regimens for mucosal visualization during sedated EGD. Methods: A double-blind randomized controlled trial was conducted at the Endoscopy Unit of Dr. Zainoel Abidin General Hospital, Banda Aceh, Indonesia, from January to December 2024. Patients scheduled for elective diagnostic EGD were randomly assigned to: Group 1 (simethicone 40 mg at 30 minutes before the procedure), Group 2 (simethicone 40 mg + 100 mL 5% sodium bicarbonate at 2 hours), Group 3 (simethicone 40 mg + N-acetylcysteine 600 mg in 100 mL water at 2 hours), or Group 4 (all three agents at 2 hours). Primary outcomes were mucosal visibility (6-site, 3-point scoring system with lower scores indicating superior mucosal visibility); procedural metrics (irrigation volume and duration); and safety (the lowest recorded SpO₂%). Data were analyzed using ANOVA or Kruskal–Wallis for continuous variables, and Chi-square or Fisher’s exact test for categorical variables, with post hoc testing as applicable. Results: A total of 168 patients were randomized into four groups (n=42 each). Groups 3 and 4 showed superior mucosal visibility compared to Groups 1 and 2 (p=0.004), with no significant difference between Groups 3 and 4. Irrigation volume differed significantly (p=0.018), lowest in Group 4. Group 3 had the shortest procedure time (3.1 ± 1.2 minutes), significantly more efficient than Groups 1 and 2, but similar to Group 4. Oxygen saturation was slightly lower in Group 3 (p<0.005), though all groups remained within safe clinical limits. Conclusions: Simethicone and N-acetylcysteine given two hours before endoscopy effectively enhanced mucosal visibility and procedural efficiency without compromising safety, offering a practical alternative to more complex regimens.
esophagogastroduodenoscopy ; mucosa visibility ; premedication ; simethicone ; N-acetylcysteine

BACKGROUND

With the increasing cancer burden in the country, nurses’ exposure to chemotherapy is inevitable as they belong to the workforce responsible for its preparation, administration, and disposal. These drugs are hazardous and necessitate special precautions to avoid direct exposure. Essentially, their competency must be aligned with the recommended safety guidelines to maintain quality patient outcomes while ensuring their safety.

The primary aim was to determine the competency level of Oncology nurses in terms of knowledge, skills, and attitude. The results were used to develop a training program framework for competency enhancement.

A descriptive correlational quantitative study was utilized. The study was conducted from December 2023 to February 2024 across three regions in Luzon, Philippines. The study included 203 Oncology nurses who fit the inclusion criteria. Data were collected via a four-part online questionnaire. Data were analyzed using frequency distribution, mean, standard deviation, and Pearson and Spearman correlation coefficients.

Oncology nurses exhibited excellent knowledge (x̄ = 16.18) and skills (x̄ = 3.86) but only fair attitudes (x̄ = 2.92). Knowledge correlated negatively with skills (ρs = -0.45, pCONCLUSIONS

Oncology nurses demonstrate comprehensive knowledge and accurate and efficient skills but maintain a neutral attitude toward handling chemotherapeutic drugs. These results relate to the complex interplay between the competency dimensions. There are still gaps and areas needing improvement that should be addressed and supported to align their competencies, especially along the skills and attitude dimensions. Training programs anchored on evidence-based practices and regulatory standards, and promoting a favorable practice environment are vital for their competency development.

OBJECTIVES

The influence of weight change on the response to neoadjuvant chemotherapy (NAC) among adult Filipino patients with breast cancer remains unclear. Currently, there has been increasing evidence that weight gain during NAC is associated with increased recurrence risk and decreased survival. This study aimed to investigate this relationship and identify significant predictors of pathologic complete response (pCR), overall survival (OS) and disease-free survival (DFS).

This is a retrospective study using data from 52 female patients who received NAC for stage II or III breast cancer and had complete records of weight before and after NAC. Significant predictors of pCR such as host factors and tumor characteristics and associations between weight change and pCR, OS and DFS were examined using univariate and multivariable logistic regression analyses.

The average weight of all patients before NAC was 57.0 kg while the average weight of all patients after NAC was 59.5 kg. The average BMI of all patients before NAC was 25.8 kg/m2. In total, 29 patients (55.8%) were classified in the overweight/obese (OW/OB) group, and the rest were classified in the normal weight/underweight (NW/UW) group. The pCR rate was 51.3% in the OW/OB group versus 48.7% in the NW/UW group (p = 0.11). Initial BMI was a significant factor for achieving pCR (hazard ratio, 3.85; 95% confidence interval [CI], 1.72-8.60, p = 0.001), suggesting that a higher initial BMI was associated with an increased likelihood of achieving pCR. Initial BMI was also an independent prognostic factor for OS (p = 0.0006) and DFS (p = 0.0005). On the other hand, no significant correlation was seen between pCR rates as well as OFS and DFS (p = 0.0551) among patients whose weight changed during the course of treatment.

These findings suggest that while initial weight may significantly predict pCR rates and affect DFS and OS, weight change during treatment may not be as influential. Further research is needed to validate these findings in more diverse and larger patient populations.

OBJECTIVES

The aim of the study is to estimate the cost of breast cancer diagnosis, treatment, and management in the Philippines. Specifically, it aims to identify the resource requirements and interventions related to breast cancer diagnosis, treatment, and management, measure resource volumes (number of units), learn to value resource items (unit costs), and determine the total cost of treatment per disease stage.

The study covered nine tertiary hospitals, seven of which were government hospitals and two were private hospitals, with all tertiary hospitals providing breast cancer services and accredited by Philippine Health Insurance Corporation (PHIC or PhilHealth) for the Z-Benefit Package. Interventions and services related to breast cancer included radiographic procedures, laboratory and imaging tests, chemotherapy drugs and medications, medical and surgical supplies, surgical rates (for breast surgery), accommodation, staff time and salary/professional fees, and other procedure fees. The study conducted in 2022, examined cost prices of breast cancer interventions and services from stage 1–3B.

Purposive and convenience sampling were used based on PhilHealth accreditation and willingness of hospitals to participate in the study. The study conducted a focus group discussion with oncologists, radiologists, anesthesiologists, and other health care providers to validate the clinical guideline used and to solicit inputs to the costing design, analysis framework, and tools for data collection. Data collection of financial cost information (charge price) was conducted using a set of costing matrices filled out by the various departments of the hospitals. Costs and median rates were calculated across hospitals on diagnostics and imaging tests, surgery costs of both public and private facilities, medical treatment, and radiotherapy.

Breast MRI, Breast Panel, and Chest CT Scan are the top 3 most expensive diagnostic procedures ranging from PhP 8,102.00 to PhP 9,800.00 per procedure. Surgical procedures for breast cancer at private hospitals and public hospitals showed huge differences in costs. The cost of a cycle of chemotherapy ranges from PhP 596.70 to PhP 3,700.00 per session, while the cost of targeted therapy can cost up to PhP 46,394.21 per session. A year of hormone therapy ranges from PhP 3,276.00 with the use of Tamoxifen, and up to PhP 68,284.00 with Goserelin. Aromatase inhibitors such as Anastrozole and Letrozole cost from PhP 18,000 to PhP 36,000, respectively. Multiple cycles depending on the diagnosis are prescribed per patient and used in combination with other chemotherapy medications or other therapies such as targeted therapy and hormone therapy are usually taken daily up to 5 to 10 years. Conventional radiotherapy can cost up to PhP 88,150.00 covering 28 sessions, CT simulation, and CT planning.

This cost study provides relevant information and better perspective on benefit development for the PHIC, policy development for Department of Health on where and how to focus their support for the patient’s financial preparedness to address medical and f inancial catastrophes.

PhilHealth needs to guide the health care providers of their costing method and to develop their own integrated, interoperable, and comprehensive cost data library.

It recommends that the government allocate budget and cover for screening and assessment for earlier stage diagnosis of patients and lower health expenditure costs on cancer treatment.

INTRODUCTION: Testicular tumours in childhood have diverse characteristics for different age ranges. This study aimed to describe the pattern, presentation and outcomes of primary testicular tumours in a paediatric population. METHODS: A retrospective study was conducted from January 2010 to December 2020 on children (≤18 years) with a diagnosis of primary testicular tumour. Baseline demographics, clinical characteristics, pathology, treatment and outcomes of these patients were analysed. The data were entered into IBM SPSS Statistics version 20.0. Chi-square test and Fisher's exact test were applied to find the statistical significance, which was set at P value ≤ 0.05. RESULTS: The study included 115 males, with 85 (73.9%) patients in the prepubertal age range with a mean age of 2.53 ± 2.06 years and 30 (26.1%) patients in the postpubertal group with a mean age of 15.73 ± 1.25 years. Yolk sac tumour was the most common (62.6%) histological subtype. Majority (46.1%) of patients had stage I disease on presentation, while 29.6% had stage IV disease. All patients underwent upfront high inguinal radical orchiectomy, which was followed by platinum-based adjuvant chemotherapy in 67% of the patients. The five-year event-free survival and overall survival for all patients were 75% and 91%, respectively. CONCLUSION Primary testicular tumours follow a bimodal age distribution pattern. Majority of patients can be cured with platinum-based chemotherapy despite having advanced disease at presentation.
Humans ; Male ; Testicular Neoplasms/mortality* ; Retrospective Studies ; Adolescent ; Child ; Child, Preschool ; Orchiectomy/methods* ; Chemotherapy, Adjuvant ; Treatment Outcome ; Neoplasm Staging ; Infant ; Endodermal Sinus Tumor/therapy* ; Neoplasms, Germ Cell and Embryonal

INTRODUCTION: Achieving low-density lipoprotein cholesterol (LDL-C) levels is key to preventing atherosclerotic cardiovascular events. However, many high-risk cardiovascular patients still experience poor LDL-C goal attainment and receive suboptimal lipid-lowering therapy (LLT) prescriptions. Herein, we evaluated LLT prescription patterns, LDL-C goal attainment and cardiovascular mortality among this population group in Singapore. METHODS: This prospective observational cohort study included 555 patients with ischaemic heart disease (IHD) admitted to the hospital in 2020. The LLT prescriptions, corresponding LDL-C levels and cardiovascular outcomes were assessed over a 24-month period. RESULTS: Most participants were male (82.3%), with 48.5% identified as Chinese. High-intensity statin prescriptions increased from 45.4% at hospital admission to 87.1% at discharge and remained stable at approximately 80% at 6, 12, and 24 months post-discharge. Combination LLT prescriptions increased from 12.3% at discharge to 33.8% by 24 months. Ezetimibe was the most commonly prescribed second-line LLT (40.8%), followed by inclisiran (1.09%) and anti-proprotein convertase subtilisin/kexin type 9 monoclonal antibody therapies (0.87%). Over 24 months, LDL-C goal attainment rates were 22.1% for LDL-C < 1.4 mmol/L and 47.2% for LDL-C < 1.8 mmol/L. Multivariable Cox proportional hazards regression indicated that achieving LDL-C < 1.8 mmol/L goal was associated with a reduction in all-cause mortality at 24 months (hazard ratio 0.53, 95% confidence interval 0.30-0.94, P = 0.030). CONCLUSION Treatment gaps in lipid management persist in 80% of the study population, indicating that statin monotherapy alone is insufficient to achieve LDL-C goals. Greater efforts to improve LDL-C goal attainment rates in high-risk cardiovascular patients are imperative.
Humans ; Male ; Cholesterol, LDL/blood* ; Female ; Myocardial Ischemia/drug therapy* ; Middle Aged ; Prospective Studies ; Aged ; Singapore/epidemiology* ; Hydroxymethylglutaryl-CoA Reductase Inhibitors/therapeutic use* ; Ezetimibe/therapeutic use* ; Anticholesteremic Agents/therapeutic use* ; Treatment Outcome

Lung cancer is the malignancy with the highest incidence and mortality burden globally, ranking first in both morbidity and mortality among all types of malignant tumors. Pathologically, lung cancer is classified into non-small cell lung cancer (NSCLC) and small cell lung cancer, with NSCLC accounting for approximately 85% of cases. Due to the often subtle or nonspecific clinical manifestations in early-stage disease, many patients are diagnosed at a locally advanced or metastatic stage, where treatment options are limited and prognosis remains poor. Therefore, molecular targeted therapy focusing on driver genes has become a key strategy to improve the survival outcomes of patients with advanced NSCLC. The epidermal growth factor receptor (EGFR) is one of the most common driver genes in NSCLC. While EGFR mutations occur in approximately 12% of advanced NSCLC patients globally, the incidence rises to 55.9% in Chinese patients. Among EGFR mutations, P-loop and αC-helix compressing (PACC) mutations account for about 12.5%. Currently, EGFR tyrosine kinase inhibitors (TKIs) have become the first-line standard treatment for advanced NSCLC patients with classical EGFR mutations, with efficacy well-established through clinical studies and real-world evidence. However, with rapid advancements in NSCLC precision medicine and deeper exploration of the EGFR mutation spectrum, EGFR PACC mutations have emerged as a key clinical focus. The structural characteristics of these mutations lead to significant variability in responses to EGFR TKIs, leaving therapeutic options still limited, while detection challenges persist due to the sensitivity constraints of current testing technologies, driving increasing demand for improved diagnostic and treatment approaches. The current clinical evidence primarily stems from retrospective analyses and small-scale exploratory studies, while prospective, large-scale, high-level evidence-based medical research specifically targeting this mutation subtype remains notably insufficient. This evidence gap has consequently led to the absence of standardized guidelines or expert consensus regarding optimal treatment strategies for advanced NSCLC with EGFR PACC mutations. As a clinical consensus specifically addressing EGFR PACC-mutant NSCLC, this document provides a comprehensive framework encompassing the clinical rationale for EGFR PACC mutation testing, therapeutic strategies for advanced-stage disease, management of treatment-related adverse events, and follow-up protocols. The consensus underscores the pivotal role of EGFR PACC mutation detection in precision medicine implementation while offering evidence-based recommendations to guide personalized therapeutic decision-making. By establishing clear clinical pathways encompassing molecular testing, therapeutic intervention, and long-term monitoring for EGFR PACC-mutant NSCLC, this consensus aims to meaningfully improve patient survival outcomes while serving as a robust, evidence-based foundation for developing personalized clinical management approaches.
Humans ; Carcinoma, Non-Small-Cell Lung/pathology* ; ErbB Receptors/antagonists & inhibitors* ; Mutation ; Lung Neoplasms/pathology* ; Protein Kinase Inhibitors/therapeutic use* ; Molecular Targeted Therapy ; Consensus

OBJECTIVE: To observe the clinical efficacy of acupuncture and moxibustion combined with medication in the treatment of functional constipation in elderly with yang deficiency and qi stagnation and its effect on emotional disorder. METHODS: A total of 86 elderly patients with functional constipation of yang deficiency and qi stagnation were randomly divided into an acupuncture and moxibustion group (43 cases, 1 case dropped out, 1 case was eliminated) and a medication group (43 cases, 1 case dropped out). The medication group was treated with lactulose oral solution, once a day；in the acupuncture and moxibustion group, on the basis of the treatment in the medication group, acupuncture at intestinal three points (bilateral Tianshu [ST25], Shangjuxu [ST37] and Guanyuan [CV4] were selected, 30 min each time, once every other day) combined with thunder-fire moxibustion (Shenque [CV8] was selected, 30 min each time, once every other day) was given. Both groups were treated for 4 weeks. Before and after treatment, the patient assessment of constipation symptoms (PAC-SYM) score, defecation situation (defecation interval time, defecation time, defecation frequency) and the scores of TCM syndrome, patient assessment of constipation quality of life (PAC-QOL), Pittsburgh sleep quality index (PSQI), self-rating anxiety scale (SAS), self-rating depression scale (SDS) were observed in the two groups. The clinical efficacy and safety of the two groups were evaluated. RESULTS: Compared before treatment, after treatment, the PAC-SYM scores, defecation interval time, defecation time, TCM syndrome scores, PAC-QOL scores, SAS scores and SDS scores in both groups were decreased (P<0.05), the defecation frequency was increased (P<0.05) in the two groups; the PSQI score in the acupuncture and moxibustion group was decreased (P<0.05). The PAC-SYM score, defecation interval time, defecation time, TCM syndrome score, PAC-QOL score, PSQI score, SAS score and SDS score after treatment in the acupuncture and moxibustion group were lower than those in the medication group (P<0.05), and the defecation frequency in the acupuncture and moxibustion group was higher than that in the medication group (P<0.05). The total effective rate of the acupuncture and moxibustion group was 92.7% (38/41), which was higher than 73.8% (31/42) in the medication group (P<0.05). The incidence of adverse reactions in the acupuncture and moxibustion group was 2.44% (1/41), while that in the medication group was 4.76% (2/42), there was no significant difference between the two groups (P>0.05). CONCLUSION Acupuncture and moxibustion combined with medication could improve the defecation situation, emotional disorder, quality of life in elderly patients with functional constipation of yang deficiency and qi stagnation.
Humans ; Moxibustion ; Constipation/drug therapy* ; Female ; Aged ; Male ; Acupuncture Therapy ; Treatment Outcome ; Qi ; Middle Aged ; Yang Deficiency/drug therapy* ; Aged, 80 and over ; Combined Modality Therapy ; Acupuncture Points