1.Key points of the International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with Achondroplasia.
Hangyu PING ; Ran DING ; Cheng HUANG ; Yue PENG ; Zikang ZHONG ; Weiguo WANG
Chinese Journal of Medical Genetics 2026;43(1):5-12
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
2.Clinical efficacy analysis of seven pediatric patients with Acute myeloid leukemia and the t(16;21)(p11;q22) FUS::ERG fusion gene.
Lihuan SHI ; Shan HUANG ; Xing XIE ; Pengkai FAN ; Haili GAO ; Yanna MAO
Chinese Journal of Medical Genetics 2026;43(2):90-95
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
3.Breast cancer in the Philippines: A financing cost assessment study.
Madeleine DE ROSAS-VALERA ; Julienne Clarize P. LECHUGA ; Lourdes Risa S. YAPCHIONGCO ; Necy S. JUAT ; Mary Juliet DE ROSAS-LABITIGAN ; Maria Lourdes E. AMARILLO ; Leo M. FLORES ; Maebel Audrey R. JOAQUIN ; Adelberto R. LAMBINICIO
Acta Medica Philippina 2025;59(17):7-15
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.
METHODSThe 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.
RESULTSBreast 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.
CONCLUSIONThis 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.
Human ; Breast Neoplasms ; Drug Therapy ; Chemotherapy ; Mastectomy ; Radiotherapy ; Radiation Therapy
4.Role of antibiotic delivery system targeting bacterial biofilm based on ε-poly- L-lysine and cyclodextrin in treatment of bone and joint infections.
Tiexin LIU ; Junqing LIN ; Xianyou ZHENG
Chinese Journal of Reparative and Reconstructive Surgery 2025;39(3):362-369
OBJECTIVE:
To explore the mechanism of antibiotic delivery system targeting bacterial biofilm with linezolid (LZD) based on ε-poly- L-lysine (ε-PLL) and cyclodextrin (CD) (ε-PLL-CD-LZD), aiming to enhance antibiotic bioavailability, effectively penetrate and disrupt biofilm structures, and thereby improve the treatment of bone and joint infections.
METHODS:
ε-PLL-CD-LZD was synthesized via chemical methods. The grafting rate of CD was characterized using nuclear magnetic resonance. In vitro biocompatibility was evaluated through live/dead cell staining after co-culturing with mouse embryonic osteoblast precursor cells (MC3T3-E1), human umbilical vein endothelial cells, and mouse embryonic fibroblast cells (3T3-L1). The biofilm-enrichment capacity of ε-PLL-CD-LZD was assessed using Staphylococcus aureus biofilms through enrichment studies. Its biofilm eradication efficacy was investigated via minimum inhibitory concentration (MIC) determination, scanning electron microscopy, and live/dead bacterial staining. A bone and joint infection model in male Sprague-Dawley rats was established to validate the antibacterial effects of ε-PLL-CD-LZD.
RESULTS:
In ε-PLL-CD-LZD, the average grafting rate of CD reached 9.88%. The cell viability exceeded 90% after co-culturing with three types cells. The strong biofilm enrichment capability was observed with a MIC of 2 mg/L. Scanning electron microscopy observations revealed the effective disruption of biofilm structure, indicating potent biofilm eradication capacity. In vivo rat experiments demonstrated that ε-PLL-CD-LZD significantly reduced bacterial load and infection positivity rate at the lesion site ( P<0.05).
CONCLUSION
The ε-PLL-CD antibiotic delivery system provides a treatment strategy for bone and joint infections with high clinical translational significance. By effectively enhancing antibiotic bioavailability, penetrating, and disrupting biofilms, it demonstrated significant anti-infection effects in animal models.
Biofilms/drug effects*
;
Animals
;
Anti-Bacterial Agents/pharmacology*
;
Polylysine/chemistry*
;
Cyclodextrins/administration & dosage*
;
Humans
;
Linezolid/pharmacology*
;
Staphylococcus aureus/physiology*
;
Rats, Sprague-Dawley
;
Mice
;
Rats
;
Male
;
Drug Delivery Systems
;
Staphylococcal Infections/drug therapy*
;
Microbial Sensitivity Tests
;
Human Umbilical Vein Endothelial Cells
;
Osteoblasts/cytology*
5.Research progress of hydrogel-based growth factors for treatment of intervertebral disc degeneration.
Chinese Journal of Reparative and Reconstructive Surgery 2025;39(11):1491-1497
OBJECTIVE:
To summarize recent research progress in hydrogel-based growth factors for treatment of intervertebral disc degeneration (IDD).
METHODS:
The relevant literature on hydrogel-based growth factors for IDD treatment at home and abroad was extensively reviewed, and their advantages and therapeutic effects in repairing IDD were analyzed and summarized.
RESULTS:
Hydrogels exhibit high hydration, biocompatibility, and biodegradability, enabling targeted delivery and sustained release of growth factors such as growth differentiation factors and transforming growth factors. This facilitates enhanced efficacy in promoting cell proliferation, extracellular matrix synthesis, and reducing inflammatory responses. Consequently, hydrogels demonstrate broad application prospects in the repair of IDD.
CONCLUSION
Research on hydrogel-based growth factors for treating IDD demonstrates advantages such as avoiding disc damage caused by repeated injections and controlling growth factor release concentrations. However, drawbacks include the limited variety of loaded growth factors and the need to verify the long-term stability and biocompatibility of hydrogels. Therefore, further research is required on aspects such as the types of loaded growth factors and the long-term stability and biocompatibility of hydrogels to establish an experimental foundation for their clinical application.
Intervertebral Disc Degeneration/therapy*
;
Hydrogels/chemistry*
;
Humans
;
Intercellular Signaling Peptides and Proteins/administration & dosage*
;
Biocompatible Materials/chemistry*
;
Animals
;
Tissue Engineering/methods*
;
Cell Proliferation/drug effects*
;
Drug Delivery Systems
6.Breast cancer in the Philippines: A financing cost assessment study
Madeleine De rosas-valera ; Julienne Clarize P. Lechuga ; Lourdes Risa S. Yapchiongco ; Necy S. Juat ; Mary Juliet De rosas-labitigan ; Maria Lourdes E. Amarillo ; Leo M. Flores ; Maebel Audrey R. Joaquin ; Adelberto R. Lambinicio
Acta Medica Philippina 2025;59(Early Access 2025):1-9
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.
METHODSThe 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.
RESULTSBreast 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.
CONCLUSIONThis 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.
Human ; Breast Neoplasms ; Drug Therapy ; Chemotherapy ; Mastectomy ; Radiotherapy ; Radiation Therapy
7.Evaluation of medication errors among inpatients in a tertiary government hospital’s pulmonary medicine service: A cross-sectional retrospective study
Judith L. Abanuales ; Jan Redmond V. Ordoñ ; ez ; Saandra Beattina B. Salandanan ; Charles Mandy G. Aryan ; Rubina Reyes-abaya
Acta Medica Philippina 2025;59(9):40-61
BACKGROUND AND OBJECTIVE
Medication errors pose substantial risks in hospitals, particularly concerning patient safety. These errors, occurring throughout the medication use process, are one of the most common causes of morbidity and mortality in clinical practice. In the Philippines, there is a lack of evidence on the prevalence and effects of medication errors, emphasizing the need for further investigation. This study evaluated the prescribing, transcribing, and monitoring errors among inpatients under the Pulmonary Medicine Service of the Department of Medicine in the Philippine General Hospital.
METHODSThis cross-sectional retrospective records review used the total population purposive sampling technique to examine eligible charts of inpatients with asthma and/or COPD from August 1 to December 31, 2022. The frequency, type, and severity of medication errors were determined. Linear regression and Cox proportional hazards models were used to examine the relationship between patient-related factors and medication errors, and length of hospital stay and mortality.
RESULTSFifty (50) out of 226 medical records were processed and analyzed. Included patients were predominantly older male adults. More than two-thirds of the patients were diagnosed with COPD while approximately one-fourth suffered from asthma. All patients were practicing polypharmacy and the vast majority presented with comorbidities. A total of 6,517 medication errors, predominantly prescribing errors (99.1%), were identified. Despite the high prevalence of medication errors, the majority were classified as “error, no harm” (98.8%), while only 1.17% were deemed as “error, harm.” As the frequency of prescribing errors increases in the power of three (rough approximation of e), from 1 to 3 to 9 to 27, etc., the expected hospital stay increases by 2.078 days (pCONCLUSION
All eligible patient charts had at least one medication error, with the majority being prescribing errors. Among the variables, prescribing errors significantly affected the length of stay, while severity of transcribing errors had a marginally significant effect. It is essential to develop comprehensive education and training initiatives and adopt a systematic approach to mitigate medication errors and promote patient safety.
Human ; Medication Errors ; Patient Safety ; Pulmonary Medicine
8.Prevalence of menopausal symptoms among young gestational trophoblastic neoplasia survivors and its relationship to their health-related quality of life
Victoria May Hembrador Velasco-redondo ; Maria Stephanie Fay Samadan Cagayan
Philippine Journal of Obstetrics and Gynecology 2025;49(2):114-120
BACKGROUND
Since the advent of chemotherapy, cure rates for gestational trophoblastic neoplasia (GTN) have improved significantly. With increased survival, patients must cope with long-term sequelae of their treatment, including early menopause. Unlike natural menopause, treatment-induced menopause may cause a sudden and dramatic decline in estrogen, which can lead to more severe symptoms.
OBJECTIVESThis study aimed to evaluate the prevalence of menopausal symptoms among young GTN survivors and to determine the impact of these symptoms on their health-related quality of life (QoL).
METHODOLOGYNinety GTN survivors (RESULTS
A total of 90 patients were enrolled in the study with a mean age of 33.06 years. Majority (81.1%) reported at least one menopausal symptom. The most prevalent symptoms were psychological symptoms, followed by somatic, then urogenital problems. Among those with an intact uterus, 8.2% reported permanent amenorrhea. Only Stage III/IV and the presence of total hysterectomy were significantly associated with menopausal symptoms. The presence of menopausal symptoms was significantly associated with poorer health-related QoL among the respondents.
CONCLUSIONMenopausal symptoms are prevalent among young GTN survivors, and these negatively affect their health-related QoL. Emphasis should be placed on recognizing and addressing these symptoms. Adjunctive procedures, especially hysterectomy, should be carefully considered because these are significantly associated with menopausal symptoms.
Human ; Chemotherapy ; Drug Therapy ; Gestational Trophoblastic Neoplasia ; Gestational Trophoblastic Disease ; Menopause
9.Competency of oncology nurses in the safe handling of chemotherapeutic drugs
Vanessa Joy P. Gasat ; Aldren R. Remon
Acta Medica Philippina 2025;59(11):63-74
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.
OBJECTIVEThe 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.
METHODSA 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.
RESULTSOncology 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.
Human ; Chemotherapy ; Drug Therapy
10.Low-density lipoprotein cholesterol goal attainment and mortality in ischaemic heart disease: a two-year observational study.
Ying Hui MAK ; Fionn CHUA ; Xuan Han KOH ; Vern Hsen TAN ; Zhong Hui LEE ; Audrey LAM ; Kim Leng TONG ; Colin YEO ; Weien CHOW ; Wann Jia LOH
Singapore medical journal 2025;66(3):154-162
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


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