Children Born Small for Gestational Age Face Higher Risk of Neurodevelopmental Disability by Age 5: Hokkaido Study

 Children born small for their gestational age (SGA) are more likely to experience neurodevelopmental difficulties by the age of five, even if they are delivered at full term, according to a new study published in Pediatrics International.

The research, led by Dr. Satoshi Suyama and colleagues from the Funded Research Division of Child and Adolescent Psychiatry, Hokkaido University Hospital, sheds light on the long-term implications of restricted fetal growth on early childhood development.
Low birth weight has long been recognized as a factor contributing to delayed growth and developmental challenges. SGA, a clinical measure used to define infants whose weight falls below the expected range for their gestational age, has been associated with a range of neurodevelopmental disorders. However, the impact of being born SGA at full term (term-SGA) has been less clear.
To explore this, the investigators utilized data from the Hokkaido Study on Environment and Children’s Health, a large prospective birth cohort. The study targeted 4,851 children who reached the age of five between April 2008 and November 2011. Mothers were asked to complete questionnaires, including the widely used Strengths and Difficulties Questionnaire (SDQ), which evaluates behavioral and emotional challenges in children. Information on birth weights was cross-referenced with data from the Japan Pediatric Society to classify children as SGA or term-SGA. Out of the invited participants, 3,484 families responded.
The analysis revealed the following findings:
  • Children born small for gestational age (SGA) showed notable differences compared to those with normal birth weight.
  • Univariate analysis revealed significant variations in hyperactivity/inattention scores and overall Total Difficulties Scores (TDS).
  • Multivariate analysis indicated that both SGA and term-SGA status were strongly linked to higher odds of neurodevelopmental disability, even after adjusting for sex and household income.
  • Children with SGA status had a 75% greater risk of elevated TDS (OR = 1.75).
  • Children born term-SGA had a 72% higher risk of elevated TDS (OR = 1.72).
  • No significant association was found between SGA status and hyperactivity or inattention scores when assessed independently.
The findings highlight that being born small for gestational age, regardless of whether a child is preterm or full term, can have lasting implications on neurodevelopment. “Our results suggest that SGA, even at term, may predispose children to difficulties in emotional and behavioral development, as reflected in the SDQ scores,” the authors noted.
The study highlights the importance of early monitoring and interventions for children born SGA, to help mitigate potential developmental challenges during the crucial early years of growth. Given the rising global focus on child health and developmental outcomes, the researchers emphasize that identifying at-risk children at birth could play a critical role in ensuring timely support and improved long-term well-being.
Reference:
Suyama, S., Itoh, M., Tamura, N., Iwata, H., Yamaguchi, T., Kobayashi, S., Miyashita, C., Saito, T., & Kishi, R. (2024). Association between small for gestational age and neurodevelopmental disability at 5 years in the Hokkaido study. Pediatrics International, 67(1), e70137. https://doi.org/10.1111/ped.70137

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Inhaled Molgramostim Promising for Autoimmune Pulmonary Alveolar Proteinosis: Phase 3 Trial Shows

USA: A phase 3 study published in the New England Journal of Medicine has highlighted encouraging results for inhaled molgramostim, a recombinant human granulocyte–macrophage colony-stimulating factor (GM-CSF), in patients with autoimmune pulmonary alveolar proteinosis (aPAP). The research was led by Dr. Bruce C. Trapnell from the Translational Pulmonary Science Center, Cincinnati Children’s Hospital Medical Center, and colleagues. 

Autoimmune pulmonary alveolar proteinosis is a rare lung disease characterized by the accumulation of surfactant within the alveoli, resulting in impaired oxygen exchange and progressive hypoxemia. The underlying cause involves autoantibodies directed against GM-CSF, a protein essential for the function of alveolar macrophages in clearing surfactant. Currently, therapeutic options remain limited, with whole lung lavage being the standard intervention.
To evaluate the efficacy and safety of inhaled molgramostim, the investigators conducted a multicenter, double-blind, placebo-controlled phase 3 trial. A total of 164 patients with confirmed aPAP were randomized to receive either molgramostim at a daily dose of 300 μg or placebo for 48 weeks. The primary endpoint assessed was the change from baseline to 24 weeks in the diffusing capacity of the lungs for carbon monoxide (DLCO), adjusted for hemoglobin and expressed as a percentage of the predicted value. Secondary endpoints included DLCO changes at 48 weeks, health-related quality of life scores measured by the St. George’s Respiratory Questionnaire (SGRQ), and exercise capacity.
Key Findings
  • At 24 weeks, patients treated with molgramostim showed a mean improvement of 9.8 percentage points in DLCO compared with 3.8 points in the placebo group. This translated into a treatment difference of 6 percentage points (95% CI, 2.5–9.4; P<0.001).
  • At 48 weeks, the molgramostim group continued to demonstrate benefit with an average DLCO increase of 11.6 percentage points versus 4.7 points in the placebo group (P<0.001).
  • Quality of life also improved, with the St. George’s Respiratory Questionnaire total score decreasing by −11.5 points at 24 weeks compared with −4.9 points in the placebo group (P=0.007).
  • No significant improvement was seen in the SGRQ activity score at 24 weeks, and therefore, no further statistical testing was conducted for subsequent secondary outcomes.
  • Safety outcomes were reassuring, with adverse events and serious adverse events occurring at similar rates in both treatment and placebo arms.
The authors concluded that once-daily inhaled molgramostim significantly improved pulmonary gas exchange compared with placebo and was well tolerated in patients with aPAP.
This trial provides the strongest evidence to date supporting inhaled GM-CSF therapy as a potential new treatment pathway for patients with autoimmune pulmonary alveolar proteinosis, a condition that currently lacks effective pharmacological options.
Reference: https://www.nejm.org/doi/abs/10.1056/NEJMoa2410542

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Low-Dose Oral Minoxidil for Hair Loss Not Linked to Tachycardia, Large Study Suggests

USA: A new retrospective study published in the Journal of the American Academy of Dermatology (JAAD) reports that low-dose oral minoxidil (LDOM), widely used off-label for nonscarring alopecia, does not increase the risk of tachycardia in patients without pre-existing cardiac conditions.

Researchers from Thomas Jefferson University’s Sidney Kimmel Medical College, led by Zachary J.K. Neubauer and colleagues, analyzed data from more than half a million patients with androgenic alopecia or other forms of nonscarring hair loss, drawn from the TriNetX database spanning 2004 to 2024. Importantly, individuals with a prior history of hypertension, tachycardia, or arrhythmia were excluded from the study.
The analysis included 524,522 patients, of which 9,267 received LDOM (≤ 5 mg/day) and the rest served as minoxidil-naïve controls. The LDOM group tended to be older (average age 45.5 years versus 39.8 years in controls) and had a higher proportion of men (33% vs. 19%). The primary outcome assessed was the incidence of tachycardia.
The key findings of the study were as follows:
  • Among those on LDOM, 126 patients (7.7%) developed tachycardia, with a median time to onset of approximately 245 days.
  • When compared to controls, the risk difference was statistically insignificant.
  • The hazard ratio (HR) for tachycardia in the LDOM group was 0.90, and after adjustments, it remained nonsignificant at 0.92.
  • Men had a 36% lower risk of developing tachycardia compared with women (HR, 0.64).
  • Asian patients also demonstrated reduced risk (HR, 0.81).
  • Conversely, White individuals were more likely to experience tachycardia compared to other groups (HR, 1.29).
  • Black individuals were also more likely to experience tachycardia compared to other groups (HR, 1.19).
According to the authors, these findings reinforce the safety of LDOM for patients without underlying cardiac issues. They noted that co-administration of beta-blockers—a practice sometimes considered to mitigate potential cardiovascular effects—is not warranted based on this evidence.
“This study represents the largest analysis to date evaluating the relationship between LDOM and tachycardia risk in nonscarring alopecia, and it is the only investigation to include a control group,” the researchers stated. However, they emphasized the need for prospective studies to validate these observations.
The findings provide reassurance for dermatologists and patients using LDOM for hair restoration, highlighting that the therapy does not appear to pose an additional risk for tachycardia when prescribed to individuals without cardiac risk factors.
Reference:
Neubauer ZJK, Lipner HI, Lipner SR. No association between low-dose oral minoxidil and tachycardia in a large retrospective cohort study of non-scarring alopecia patients. J Am Acad Dermatol. 2025 Aug 8:S0190-9622(25)02587-3. doi: 10.1016/j.jaad.2025.07.065. Epub ahead of print. PMID: 40784566.

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Keeping It Warm: A Comprehensive Study on Postoperative Hypothermia in Non-Cardiac Surgery

Postoperative hypothermia is a prevalent issue among non-cardiac surgery patients that can have serious consequences but often goes unaddressed. To address this problem, a study was conducted to identify risk factors for postoperative hypothermia in this patient population. Recently published study analyzed various factors contributing to postoperative hypothermia, focusing on age, BMI, ASA classification, type of surgery, blood loss, fluid administration, blood transfusion, anesthesia and surgery duration as potential risk factors.

Risk Factors for Postoperative Hypothermia

The analysis revealed that age over 60 years, BMI less than 18.5 kg/m², ASA III-IV classification, endoscopic surgery, blood loss exceeding 100 ml, intravenous fluid administration over 1000 ml, blood transfusion, anesthesia time longer than 60 minutes, and surgery time exceeding 60 minutes were associated with an increased risk of postoperative hypothermia. Elderly patients are particularly vulnerable due to reduced subcutaneous fat and thermoregulatory function. Patients with high body fat are at a lower risk due to increased metabolic rate, while those with low BMI are more prone to hypothermia. The study emphasized the importance of proper temperature management for elderly and high-risk patients. It highlighted the impact of longer anesthesia and surgery times on hypothermia risk, suggesting that prolonged exposure to anesthetic drugs and low-temperature environments can lead to heat loss. Endoscopic surgeries were found to increase the risk of hypothermia due to continuous insufflation and irrigation, leading to heat loss. Fluid rehydration exceeding 1000 ml and blood loss over 100 ml were also identified as significant predictors of hypothermia. Despite the comprehensive analysis, the study acknowledged limitations such as potential language bias, incomplete data, and a focus on adult non-cardiac surgery patients. The findings emphasize the need for tailored interventions and proactive measures to prevent postoperative hypothermia in at-risk populations. The quality of evidence for identified risk factors varied from very low to low, with intraoperative blood loss emerging as a key predictor. The study underscores the importance of early identification and management of high-risk individuals to mitigate complications and enhance patient outcomes following non-cardiac surgeries.

Conclusion

In conclusion, this research provides valuable insights into risk factors for postoperative hypothermia in non-cardiac surgery patients, offering a foundation for developing effective management protocols. Recognizing and addressing these risk factors early on can significantly improve patient outcomes and reduce the incidence of postoperative hypothermia in this population.

Key Points

– Age over 60 years, BMI less than 18.5 kg/m², ASA III-IV classification, endoscopic surgery, blood loss exceeding 100 ml, intravenous fluid administration over 1000 ml, blood transfusion, anesthesia time longer than 60 minutes, and surgery time exceeding 60 minutes were identified as risk factors for postoperative hypothermia.

– Elderly patients are more vulnerable to hypothermia due to reduced subcutaneous fat and thermoregulatory function, while patients with high body fat have a lower risk because of an increased metabolic rate.

– Prolonged anesthesia and surgery times contribute to hypothermia risk by exposing patients to heat loss in low-temperature environments.

– Endoscopic surgeries increase the risk of hypothermia through continuous insufflation and irrigation, leading to heat loss. – Factors like fluid rehydration exceeding 1000 ml and blood loss over 100 ml were significant predictors of postoperative hypothermia.

– The study highlights the importance of tailored interventions and proactive measures to prevent postoperative hypothermia in high-risk populations, emphasizing the need for early identification and management of at-risk individuals to enhance patient outcomes following non-cardiac surgeries.

Reference –

Ruyi Tan et al. (2025). Risk Factors For Postoperative Hypothermia In Non-Cardiac Surgery Patients: A Systematic Review And Meta-Analysis. *BMC Anesthesiology*, 25. https://doi.org/10.1186/s12871-025-03089-9.

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Pediatric Pain Relief: The Role of Regional Anesthesia in Renal Disease Management, study finds

Renal disease in pediatric patients presents significant challenges, particularly in anesthetic management, due to its association with high morbidity and mortality rates. Achieving effective perioperative pain management is crucial, and pediatric regional anesthesia emerges as a valuable strategy to enhance pain control. Recent study explores the impact of ultrasound-guided supraclavicular brachial plexus block (BPB) as an adjunct to sevoflurane anesthesia during brachiobasilic arteriovenous fistula (AVF) operations in children with end-stage renal disease (ESRD).

Challenges in AVF Creation and the Role of BPB

The creation of AVF is critical for hemodialysis access, yet the procedure faces complications, including a high primary failure rate largely attributed to inadequate vessel dilation and sympathetic overflow-induced vasospasm. The implementation of BPB can induce sympathetic blockade, leading to vessel dilation and improved blood flow—key factors in ensuring successful AVF maturation.

Study Design and Objectives

In a randomized controlled trial, 60 pediatric patients with ESRD participated, divided into two groups receiving either general anesthesia with BPB or general anesthesia with saline injection. The study aimed to measure the time to AVF maturation, hemodynamic stability during surgery, the incidence of emergence agitation, and postoperative analgesia.

Results and Clinical Implications

Results indicated that patients receiving BPB demonstrated significantly improved outcomes: faster maturation of the AVF (mean of 12 weeks compared to 15 weeks in the control group) and enhanced hemodynamics, manifested as lower mean arterial pressure (MAP) reduction and heart rates post-block. Notably, the basilic vein diameter post-block was greater in the BPB group, suggesting improved conditions for AVF creation and maturation. Additionally, instances of emergence agitation were reduced from 66% in the control group to 20% in the BPB group, indicating a smoother recovery phase. Pain scores at varying time intervals after surgery were also significantly lower for those receiving BPB, demonstrating effective analgesia.

Conclusion and Future Directions

This trial underscores the role of regional anesthesia as a feasible approach in pediatric patients undergoing AVF surgery. By fostering vasodilation and facilitating adequate blood flow, BPB not only reduces the chances of AVF failure but also enhances the overall surgical experience through better hemodynamic management and reduced perioperative discomfort. The findings advocate for the integration of ultrasound-guided regional anesthesia techniques into the standard anesthetic regimen for pediatric patients with ESRD, aiming for improved surgical outcomes and patient comfort. Future research is encouraged to further explore long-term effects and validate these findings across larger cohorts.

Key Points

– Pediatric renal disease complicates anesthetic management due to increased morbidity and mortality, necessitating effective perioperative pain control strategies, such as pediatric regional anesthesia.

– The study focuses on ultrasound-guided supraclavicular brachial plexus block (BPB) as an adjunct to sevoflurane anesthesia for improving outcomes during brachiobasilic arteriovenous fistula (AVF) surgeries in children with end-stage renal disease (ESRD).

– In a randomized controlled trial involving 60 pediatric ESRD patients, two groups underwent either general anesthesia with BPB or with saline injection, assessing AVF maturation time, hemodynamic stability, emergence agitation, and postoperative analgesia.

– BPB was associated with significantly faster AVF maturation (12 weeks vs. 15 weeks in the control group), better hemodynamic parameters, and a larger basilic vein diameter, which are vital for successful AVF creation.

– Emergence agitation was markedly reduced in the BPB group (20%) compared to the control group (66%), indicating improved recovery experiences, along with significantly lower pain scores at various postoperative intervals.

– The findings support the use of regional anesthesia techniques like BPB in pediatric patients with ESRD undergoing AVF surgery, emphasizing potential benefits in surgical outcomes and patient comfort while highlighting the need for further research to confirm these results in larger populations.

Reference –

S. Elsawy et al. (2025). Ultrasound-Guided Supraclavicular Brachial Plexus Block As An Additive To Sevoflurane Anesthesia In Pediatrics Undergoing Brachiobasilic Arteriovenous Fistula Operation: Randomized Controlled Clinical Trial. *BMC Anesthesiology*, 25. https://doi.org/10.1186/s12871-025-03091-1.

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From Data to Action: Study Identifying Determinants of Short Birth Intervals

Recent study focused on predicting short birth intervals (defined as less than 33 months) among reproductive-age women in East Africa, using supervised machine learning (ML) models to identify key determinants. Utilized recent Demographic and Health Surveys (DHS) data from 11 East African countries, including Uganda and Ethiopia, with a sample size of 100,246 women who had at least two consecutive live births. Implemented a two-stage stratified cluster sampling technique to ensure representation of both urban and rural populations. Employed various techniques, including data cleaning, normalization, feature selection through Recursive Feature Elimination (RFE), and handling missing data via mode imputation. Continuous variables were discretized for improved model interpretability. Utilized Python libraries (Pandas, scikit-learn) to build models, including Random Forest (RF), Decision Tree (DT), Logistic Regression (LR), and Naive Bayes (NB). The dataset was split into training (80%) and testing (20%) sets for evaluation. The Random Forest model achieved the highest accuracy (79.4%), precision (79.0%), recall (91%), and F1-score (84%), outperforming DT and LR. Important factors included: – -Age-: Women aged 15-24 had higher risks of short birth intervals. Higher risks were associated with having 2-3 children. Women from poorer households exhibited increased rates of short intervals. Lack of education correlated with shorter birth spacing. Limited access and exposure influenced family planning decisions. The reliance on self-reported data from DHS, which could lead to potential biases. The findings may not fully represent populations in regions not included in the study. Although supervised ML models were effective, the inherent complexity might not be easily interpretable for public health practitioners. The study successfully applied machine learning to predict short birth intervals and uncover significant determinants, emphasizing the need for enhanced family planning services and maternal education in East Africa. Recommendations included integrating ML models into public health strategies to inform policy-making and improve maternal and child health outcomes. The research underscores the importance of addressing underlying socioeconomic factors to improve birth spacing practices.

Key Points

– -Data Utilization and Methodology-: Analyzed data from Demographic and Health Surveys (DHS) across 11 East African countries, focusing on a robust sample of 100,246 women with at least two consecutive live births. A two-stage stratified cluster sampling ensured diverse representation from urban and rural areas.

– -Data Processing Techniques-: Implemented comprehensive data preprocessing methods, including cleaning and normalization, feature selection via Recursive Feature Elimination (RFE), and mode imputation for missing values. Continuous variables were transformed for better model interpretability.

– -Modeling Approach-: Employed various supervised machine learning algorithms such as Random Forest, Decision Tree, Logistic Regression, and Naive Bayes, utilizing Python libraries for model development. The dataset was partitioned into training (80%) and testing (20%) sets, facilitating thorough evaluation of model performance.

– -Performance Metrics-: The Random Forest model demonstrated superior efficacy, achieving an accuracy of 79.4%, precision of 79.0%, recall of 91%, and an F1-score of 84%, surpassing the performance of the other modeling approaches.

– -Determinants of Short Birth Intervals-: Identified critical factors influencing short birth intervals, highlighting that younger women (15-24 years), those with 2-3 children, lower socioeconomic status, lack of maternal education, and restricted access to healthcare and media exposure were significantly associated with increased risks of shorter birth spacing.

– -Recommendations and Policy Implications-: Encouraged the integration of machine learning techniques into public health frameworks to enhance family planning services and promote maternal education. The study emphasized addressing socioeconomic factors to improve birth-spacing practices and overall maternal and child health outcomes in East Africa.

Reference –

Tirualem Zeleke Yehuala et al. (2025). Exploring Machine Learning Algorithms To Predict Short Birth Intervals And Identify Its Determinants Among Reproductive-Age Women In East Africa. *BMC Pregnancy And Childbirth*, 25. https://doi.org/10.1186/s12884-025-07668-z.

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NEET MDS: KNRUHS to end Online Registration After Cut-Off Reduction, details

Telangana- The Kaloji Narayan Rao University of Health Sciences (KNRUHS) has issued a notification regarding the online registration after the reduction of the cut-off score for the Master of Dental Surgery (MDS) admission under the Competent Authority and Management Quota for the academic year 2025-26.

KNRUHS has notified on the online registration from NEET-MDS- 2025 qualified candidates for admission into MDS courses under Competent Authority into Dental Colleges and Management Quota MQ1, MQ2 (NRI), MQ3 categories for the academic year 2025-26 into affiliated Private Dental colleges in Telangana State including Army College of Dental Sciences.

Also Read: MD Paediatrics medico denied exam eligibility due to maternity leave- Telangana HC seeks response from NMC, KNRUHS

Notification is issued for online registration for web-based counselling to update the State Merit position for admission into Competent Authority Quota and Management Quota seats only. The Updated Final Merit list will be notified after scrutiny of all certificates uploaded at the time of online registration. The total number of vacant seats available under the Competent Authority Quota and the Management Quota for the academic year 2025-26 will be notified on the KNRUHS website before exercising web options for counselling. Tuition fee payable will be as notified by the Government of Telangana.

REVISED CUT-OFF SCORE IN NEET-MDS-2025 EXAM FOR MDS COURSES

Candidates should have secured the following cutoff score or above in NEET-MDS-2025-

S.NO

CATEGORY

MINIMUM ELIGIBILITY CRITERIA

REVISED CUT-OFF SCORE

1

General Category

30.137 Percentile 197

168

2

General – PWD

25.137 Percentile

182

3

SC/ST/OBC (including PWD of SC/ST/OBC)

20.137 Percentile

168

Meanwhile, as per the notification, the candidates can register online and upload scanned certificates on the official website of KNRUHS from 4.00 PM on 21 August 2025 up to tomorrow, i.e. 23 August 2025, 01.00 PM.

Provisional Final Merit position of the applied candidates will be prepared based on NEET-MDS–2025 Rank and other eligibility criteria notified hereunder. However, candidates who have already registered previously under the Competent Authority and the Management Quota need not register again.

To view the notifications, click the link below

https://medicaldialogues.in/pdf_upload/notification-1-298469.pdf

https://medicaldialogues.in/pdf_upload/notification-2-298470.pdf

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Ravenbhel’s Bilastine, Montelukast FDC Under Review, CDSCO Panel Seeks Pediatric Expert Opinion

New Delhi: In response to a proposal submitted by Ravenbhel Healthcare of the fixed dose combination (FDC) pulmonary drug Bilastine 10 mg plus Montelukast Sodium IP eq. to Montelukast 4 mg per 5 mL oral solution, the Subject Expert Committee (SEC) under the Central Drugs Standard Control Organisation (CDSCO) has recommended inviting a pediatrician for a detailed discussion on the matter.

This came after the firm presented the proposal along with justification for BE and Phase III CT waiver before the committee.

Bilastine is an antihistamine medication used to treat hives (urticaria), allergic rhinitis and itchy inflamed eyes (allergic conjunctivitis) caused by an allergy. It is a second-generation antihistamine and takes effect by selectively inhibiting the histamine H1 receptor, preventing these allergic reactions.

Montelukast is in a class of medications called leukotriene receptor antagonists (LTRAs). It works by blocking the action of substances in the body that cause the symptoms of asthma and allergic rhinitis.

Montelukast is used to prevent wheezing, difficulty breathing, chest tightness, and coughing caused by asthma in adults and children 12 months of age and older. Montelukast is also used to prevent bronchospasm (breathing difficulties) during exercise in adults and children 6 years of age and older.

At the recent SEC meeting for Pulmonary held on the 7th August 2025, the expert panel reviewed the proposal along with justification for BE and Phase III CT waiver before the committee.

After detailed deliberation, the committee opined that Pediatrician may be invited in next meeting for wider discussion in the matter.

Also Read: Sun Pharma’s Dapagliflozin, Glimepiride, Metformin FDC Fails to Secure CDSCO Panel Nod Over Safety, Utility Concerns

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Low daily fluid intake linked to higher stress hormone response in adults

People who drink less than the recommended daily fluid intake experience a greater stress hormone response, which is associated with an increased risk of heart disease, diabetes and depression, according to a new study from scientists in Liverpool, U.K.

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Merck’s SpringWorks Therapeutics gets nod for Ogsiveo from European Commission for adults with Desmoid Tumors

Stamford: SpringWorks Therapeutics, Inc., a healthcare company of Merck, has received marketing authorization from the European Commission for OGSIVEO (nirogacestat), an oral gamma secretase inhibitor, as monotherapy for the treatment of adults with progressing desmoid tumors who require systemic treatment. OGSIVEO is a therapy approved in the European Union (EU) to treat desmoid tumors.

“Desmoid tumors can have a profound impact on people’s lives and are difficult to manage due to their invasive nature and high rates of recurrence. Until now, there have been no approved medicines in Europe,” said Bernd Kasper, M.D., Ph.D., Professor, University of Heidelberg, Mannheim Cancer Center, Mannheim, Germany, and principal investigator of the DeFi trial. “OGSIVEO is a highly innovative therapy with efficacy data demonstrating both meaningful antitumor activity and a significant improvement in desmoid tumor symptoms, including a significant reduction in pain which is the most debilitating symptom reported by patients.”

“This approval is a long-awaited advance for desmoid tumor patients, their families and physicians in Europe,” said Lynne Hernandez, Executive Director of the Desmoid Tumor Research Foundation. “It is our hope that patients will benefit from greater awareness of desmoid tumors, faster diagnoses, and better outcomes now that there is an approved treatment.”

Desmoid tumors are rare, locally aggressive tumors that form in the connective tissues of the body. Approximately 1,300 to 2,300 new cases of desmoid tumors are diagnosed annually in the EU. These tumors can cause severe pain, limited function, loss of mobility, disfigurement and fatigue. They are challenging to manage because of their unpredictable nature and high rate of recurrence, which can significantly impact an individual’s quality of life. Desmoid tumor experts and treatment guidelines now recommend medical therapy as first-line intervention instead of surgery for most tumor locations requiring treatment.

“We would like to extend our gratitude to the patients, families, investigators, and advocacy organizations who helped make this EC approval possible,” said Danny Bar-Zohar, MD, CEO of Healthcare and Executive Board Member at Merck KGaA, Darmstadt, Germany. “OGSIVEO is already established as the standard of care systemic therapy for desmoid tumors in the U.S., and our goal is to bring the same treatment benefits to patients in Europe. Following last month’s EC approval of our therapy for patients with NF1-PN, we are in the unique position of launching two innovative treatments — underscoring our commitment to the rare tumor patient community.”

The EC approval of OGSIVEO is based on results from the Phase 3 DeFi trial, which enrolled 142 adult patients with progressing desmoid tumors and met the primary endpoint of improving progression-free survival (PFS). OGSIVEO demonstrated a statistically significant improvement over placebo with a 71% reduction in the risk of disease progression (hazard ratio (HR) = 0.29 (95% CI: 0.15, 0.55); p< 0.001). OGSIVEO also demonstrated a significant improvement in objective response rate (ORR). The confirmed ORR based on RECIST v1.1 was 41% with OGSIVEO versus 8% with placebo (p<0.001); the complete response rate was 7% in the OGSIVEO arm and 0% in the placebo arm. The median time to first response was 5.6 months with OGSIVEO and 11.1 months with placebo. Additionally, OGSIVEO demonstrated early and sustained improvement in patient-reported outcomes (PROs), including pain (p<0.001), desmoid tumor-specific symptoms (p<0.001), physical/role functioning (p<0.001), and overall health-related quality of life (p≤0.01).

OGSIVEO exhibited a manageable safety and tolerability profile.

The FDA and the EMA have granted Orphan Drug designation for OGSIVEO for the treatment of desmoid tumors.

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