Single Center Experience Research Articles

Real-world clinical outcomes with fostamatinib for the treatment of refractory chronic immune thrombocytopenia: a single-center experience.

Fostamatinib is a spleen tyrosine kinase inhibitor indicated for the treatment of chronic immune thrombocytopenia (ITP) unresponsive to a previous treatment. Real-world studies evaluating the utilization and effectiveness of fostamatinib outside the context of a clinical trial are lacking. The objective of this analysis was to evaluate the effectiveness of fostamatinib for the treatment of ITP in a real-world cohort. We conducted a single-center, retrospective, observational study to evaluate the effectiveness of fostamatinib for the treatment of ITP. The primary endpoint was durable response as defined by the American Society of Hematology ITP response criteria. Secondary endpoints included overall response rate, time to response, and safety. Subgroup analysis was performed to assess frequency of durable response in key subgroups of patients based on prior therapies. Thirty-one patients treated with fostamatinib for ITP were included in our analysis. Patients had received a median of four prior lines of therapy. Ten patients (32%) achieved a durable response. Most durable responders maintained their response at 24 months (n = 7; 70%). The median time to response was 9 days. Four patients (13%) discontinued fostamatinib due to an adverse event. Subgroups who had higher rates of durable responses included those who had received two to three prior lines of therapy (40%), splenectomized patients (50%), and those who had not received prior rituximab (55%). Fostamatinib therapy in a real-world population of patients with heavily pretreated ITP led to a durable response in a third of patients, which was maintained for most responders.

The Effect of Age on High-Dose Therapy with Autologous Stem Cell Support in Multiple Myeloma: A Single-Center Experience

Background: This retrospective one-center study demonstrates the complications related to high-dose therapy with autologous stem cell support (HDT) and the survival of multiple myeloma (MM) patients according to age groups. Methods: We categorized the patients into two groups: those who were ≤65 years old (group 1) (N = 115) and those who were >65 years old (group 2) (N = 26). The mean duration of follow-up was 48 (1–125) months. Results: In group 2 patients, the use of a reduced dosage of melphalan (12 [46%] versus 30 [26%]) was more frequent in comparison to group 1 (p = 0.046). There was a statistically significant difference between the two groups regarding the neutrophil engraftment days (p = 0.001) and the median progression-free survival (PFS) (p = 0.02). The PFS was 44 months for group 1 and 30 months for group 2. There was no statistically significant difference between the groups in relation to the median duration of hospitalization, presence of bacteremia, intravenous antibiotic administration, and overall survival (OS). Conclusions: The study’s results indicate that HDT is a reliable method of treatment for older patients with MM, provided that they obtain a suitable conditioning regimen and, furthermore, these patients achieved a comparable OS rate to that of younger patients.

Concurrent Use of Trastuzumab Deruxtecan and Radiation Therapy in HER2-positive and HER2-low Metastatic Breast Cancer: A Single-center Experience and Review of the Literature.

Recent DESTINY-Breast trials have demonstrated trastuzumab deruxtecan's effectiveness in HER2-positive and HER2-low metastatic breast cancer. However, safety concerns remain regarding its combination with radiation therapy (RT). The purpose of this work is to assess the toxicity profile of combining trastuzumab deruxtecan and RT in patients with HER2-positive and HER2-low metastatic breast cancer to address these concerns. We conducted a retrospective study which included patients treated at Institut Curie Paris between November 2020 and January 2024. Patients with HER2-positive and HER2-low metastatic breast cancer who received concurrent trastuzumab deruxtecan and RT were identified. Data on patient demographics, treatment regimens, radiation doses, toxicity profiles, and treatment discontinuations were collected. Follow-up was conducted from the last day of radiotherapy until death or the last examination and toxicities were graded using the CTCAE V5.0. The studied population includes all 33 patients with HER2-positive and HER2-low metastatic breast cancer who underwent concurrent treatment with trastuzumab deruxtecan and radiotherapy. The median follow-up was 11 months. The most common acute grade 1 toxicity was nausea. Grade 2 toxicities affected 21.2% of patients, including asthenia, mucositis, cardiac decompensation, and diarrhea. Trastuzumab deruxtecan discontinuation occurred in 5 patients due to systemic treatment-related toxicities, including nausea, thrombocytopenia, neutropenia, and cardiac decompensation. There were 21.2% reported with late toxicities, with nausea being the most prevalent. Our series of patients who received concurrent treatment of radiotherapy and trastuzumab deruxtecan are showing acceptable toxicity. Larger prospective studies are needed to evaluate the toxicity and efficacy of this combination.

Clinical Profile and Outcome of Patients Presenting With Acute-on-Chronic Liver Failure: A Single-Center Experience

Clinical Profile and Outcome of Patients Presenting With Acute-on-Chronic Liver Failure: A Single-Center Experience

Real-world use of imipenem/cilastatin/relebactam for the treatment of KPC-producing Klebsiella pneumoniae complex and difficult-to-treat resistance (DTR) Pseudomonas aeruginosa infections: a single-center preliminary experience

IntroductionReal-life experience with imipenem/cilastatin/relebactam (IMI/REL) for the treatment of KPC-producing Klebsiella pneumoniae complex (KPC-Kp) and difficult-to-treat resistance (DTR) Pseudomonas aeruginosa (DTR-PA) infections is herein described.MethodsAdult patients with KPC-Kp or DTR-PA infections who received ≥48 h of IMI/REL were included. Clinical and microbiological outcomes were retrieved through the medical records. Primary outcome was clinical cure. Secondary outcomes included mortality from infection onset and adverse effects attributable to IMI/REL.ResultsWe included 10 patients with different infections caused by DTR-PA (n = 4), KPC-Kp [n = 5, of which 3 ceftazidime/avibactam-resistant (CTV-R KPC-Kp), 2 CTV susceptible (CTV-S KPC-Kp)] or both DTR-PA/KPC-Kp (n = 1) successfully treated with IMI/REL: 3 hospital-acquired pneumonia, 1 ventilator-associated pneumonia, 2 skin and soft tissue infections, 1 osteomyelitis, 2 bloodstream infections, 1 complicated urinary tract infection. Clinical cure was achieved in all cases. No patients died and no side effect were reported.DiscussionWe reported the preliminary real-life experience on the successful and safe use of IMI/REL for the treatment of KPC-Kp or DTR-PA complicated infections, including pneumonia and bone infections.

The efficacy and safety of second salvage autologous transplantation in myeloma patients

Despite the availability of many novel therapies for multiple myeloma, it remains an incurable disease with relapse fated in almost all patients. In the era of modern agents, second autologous stem cell transplantation still holds its role in patients relapsing after first-line autologous transplant. The authors reviewed a single-center experience with a second auto-SCT for relapsed multiple myeloma. Thirty patients had received a salvage auto-SCT at the institution. The median follow-up after diagnosis was 86 months, and the median time between transplants was 59.1 months. Response before second ASCT was the following: CR – 11 cases, VGPR – 9 cases, PR – 10 cases. Most patients received reduced dose (140 mg/m2) of melphalan as a conditioning regimen for the second auto-SCT. Treatment-related mortality was 3%. With a median follow-up time of 34 months after the second transplant, median progression-free survival was 24 months. The median PFS in the patients achieving CR or VGPR at day 100 after the second transplantation was 32 months. By 15 months, all patients achieved only partial remission progressed, with a median PFS of 8.5 months. During the follow-up period, no MDS or AML developed, and the frequency of second malignancy was also low, 3%. In conclusion, second autologous stem cell transplantation is a well-tolerated and effective treatment option for relapsed multiple myeloma in selected patients, though with a shorter PFS than in first remission.

Culture-proven Bacterial Conjunctivitis in Newborns: Five-year Single-center Experience

Culture-proven Bacterial Conjunctivitis in Newborns: Five-year Single-center Experience

Impact of Frailty on Functional Improvement Following Traumatic Spinal Cord Injury: A Japanese Single-Center Experience

Study Design: This is a retrospective case series study. Objective: The aim of this study was to investigate whether frailty contributes to functional recovery in individuals with spinal cord injury (SCI). Methods: A total of 121 patients with SCI (106 cervical SCI, 15 thoracic SCI) discharged from our center over the past three years were studied. Moreover, 11-factor modified frailty index (mFI) scores, the length of hospital stays, the rate of returning home, and improvement in Spinal Cord Independence Measure (SCIM) scores were assessed retrospectively. Results: The average age at the time of injury for all 121 cases was 59.6 years. Based on pre-injury assessments, 24 cases were categorized as the Frail group, and 97 cases were categorized as the Robust group. The Frail group had SCIM improvement rates of 16.7% and a home discharge rate of 45.8%. In contrast, the Robust group had SCIM improvement rates of 33.5% and a home discharge rate of 68.0%, with statistically significant differences between the two groups. A significant negative correlation was observed between mFI scores and SCIM improvement rates (R = −0.231, p = 0.014). Conclusions: This study suggests that individuals with pre-existing frailty before SCI experience poorer SCIM improvement rates and face challenges in returning home.

Gastrointestinal system involvement in patients with primary immunodeficiency: a single center experience

Aim Primary immunodeficiencies (PIDs) are a heterogeneous disorder group characterized by an impaired immune system, leading to an increased susceptibility to infections and a wide range of clinical manifestations, including gastrointestinal (GI) complications. This study aimed to assess the GI manifestations of PID patients and highlight the significance of atypical gastrointestinal symptoms in the early diagnosis of these patients. Methods A retrospective analysis was conducted on pediatric patients diagnosed with PIDs at Selcuk University Medical Faculty from 2011 to 2021. The study focused on demographic data, clinical presentation, genetic mutations, and GI manifestations, including endoscopic evaluation. Patients were categorized according to the International Union of Immunological Societies (IUIS) PID classifications. Statistical analyses were performed to identify significant associations between PID types and GI manifestations. Results The cohort comprised 101 patients, with 46% presenting with GI symptoms, including malnutrition and chronic diarrhea, as the most common findings. Primary antibody deficiency (PAD) emerged as the most prevalent PID with GI involvement, followed by combined immunodeficiencies (CID) with associated or syndromic features. Endoscopic evaluations revealed inflammatory bowel disease (IBD)-like colitis in a significant subgroup of patients. The analysis showed that some GI symptoms were more common in specific PID categories, highlighting the importance of early gastroenterological assessment in PID patients. Conclusion Recognition of common GI symptoms in pediatric patients with PIDs may facilitate early diagnosis and prompt multidisciplinary management, potentially improving patient outcomes. The study highlights the necessity of considering PIDs in diagnosing persistent or severe GI symptoms in children.

Clinical Characteristics and Visual Prognosis of Idiopathic Intracranial Hypertension in Korea: A Single-center Experience

Purpose: To examine the clinical characteristics, demographic features, and visual prognosis of idiopathic intracranial hypertension (IIH) in Korean patients.Methods: We conducted a retrospective chart review of patients diagnosed with IIH who visited the Neuro-ophthalmology Clinic at Samsung Medical Center from September 2011 to January 2023. This analysis focused on demographic characteristics at onset, clinical manifestations, and visual outcomes at the last follow-up.Results: Out of 34 patients, 20 met the modified Dandy criteria and 14 were presumed to have IIH. The mean age at onset was 32.97 ± 9.6 years with females comprising 85.3% (n = 29) of the sample. The average body mass index (BMI) was 29.15 ± 6.43, with 47.0% of patients (n = 16) classified as obese. The most frequent symptom was headache, reported by 85.3% (n = 29) of patients, followed by pulsatile tinnitus (64.7%, n = 22) and transient visual obscuration (TVO; 32.4%, n = 11). Treatment included observation for 3 patients (8.8%), acetazolamide therapy for 26 patients (76.5%), and ventriculo-peritoneal shunt (V-P shunt) for 2 patients (5.9%). Out of 23 patients with follow-up > 6 months, 87.0%, 8.7%, and 4.3% showed good, fair, and poor visual outcomes, respectively; optic nerve atrophy was observed in 19.6% (9 eyes). The initial peripapillary retinal nerve fiber layer (pRNFL) thickness measured by OCT was 143.56 ± 62.41 μm in the right eye and 139.3 ± 76.38 μm in the left which decreased to 97.39 ± 25.72 and 94.91 ± 19.32 μm, respectively by the last examination.Conclusions: While the proportion of obese patients in this Korean cohort and the average BMI were lower than in western countries, both have increased relative to previous reports from Korea. Most patients experienced a benign course.

Long-Term Results of Percutaneous Balloon Aortic Valvuloplasty in Children With Aortic Stenosis: A Single-Center Experience

Long-Term Results of Percutaneous Balloon Aortic Valvuloplasty in Children With Aortic Stenosis: A Single-Center Experience

Survival and prognostic factors of primary retroperitoneal sarcomas after surgery: a single-center experience.

The percentage of retroperitoneal sarcomas (RPS) among all soft tissue sarcomas ranges from 10 to 15%. Surgery remains the gold standard for RPS. In this study, we analyzed the impact of surgical treatment for primary RPS on recurrence and overall mortality at a Chinese institution and identified and evaluated prognostic variables. Data from patients with RPS who underwent surgical treatment were retrospectively analyzed. The patients were treated at a single center from January 2000 to June 2018. Retrospectively collected demographic, clinicopathological, and surgical factors were examined. Overall survival (OS) and disease-free survival (DSF) were used as the primary endpoints. Predicted 5-year survival rates, encompassing both DFS and OS, were derived from the Sarculator prognostic nomogram. A total of 110 patients met the inclusion criteria. The median follow-up time after surgery for patients with primary RPS was 5.3 years. During this period, 59 patients died. The 5-year OS and DFS estimates were 63.5% and 35.3%, respectively. In a multivariate analysis, poor OS following surgical treatment of primary RPS was independently correlated with FNCLCC grade (p < 0.001) and surgical margin status (p = 0.016). FNCLCC grade (p = 0.001) and surgical margin status (p = 0.002) were also independently associated with poor DFS. The C-indices for 5-year OS and DFS survival utilizing the Sarculator prognostic nomogram were 0.71 and 0.73 respectively. The overall mortality rate of patients with RPS was considered acceptable. OS and DFS prognostic markers were established for primary RPS. Tumor grade and intraregional margins are other factors that affect survival and recurrence.

Endoscopic resection for gastric submucosal tumors: A single-center experience in Japan.

Endoscopic resection (ER) for gastric submucosal tumors (SMTs) has gained prominence in recent years, with studies emerging from various countries. However, there is a paucity of reports from Japan. We aimed to elucidate the efficacy and safety of ER for gastric SMT in Japan. In this retrospective observational study, we investigated the outcomes of consecutive patients who underwent ER for gastric SMT from January 2017 to May 2023. The outcome variables assessed included the complete resection rate, procedure time, closure-related outcomes, and the incidence of adverse events. A total of 13 patients were included in the analysis. The median procedure time was 163 (55-283) min. Complete full-thickness resection was performed in seven cases, while in four cases, the serosa remained, and in two cases, the outer layer of the muscularis propria remained. In two cases where the SMT was located on the anterior side, conversion to laparoscopic surgery became necessary, resulting in a procedural success rate of 84.6% (11/13). Excluding these two cases, endoscopic closure of the defect was successfully accomplished in the remaining 11 cases. R0 resection was achieved in 12 out of 13 cases (92.3%). Although one patient had peritonitis, which was successfully treated conservatively, no other treatment-related adverse events were encountered. Although ER for SMT on the anterior side may be challenging, our experience revealed that ER is a safe and efficacious approach for gastric SMT.

Intellectual Disabilities and Neurocognitive Impairment in Adult Patients with Inherited Metabolic Diseases: A UK Single Centre Experience

Inherited metabolic diseases (IMDs) are a group of heterogeneous genetic disorders resulting in substrate accumulation, energy deficiency, or complex molecular defects due to the failure of specific molecules to act as enzymes, cofactors, transporters, or receptors in specific metabolic pathways. The pathophysiological changes seen in IMDs are sometimes associated with intellectual disability (ID) or neurocognitive decline, necessitating multidisciplinary input. We here describe our experience at one tertiary metabolic centre in the UK. We reviewed the case prevalence and existing service provision in one adult IMD service covering a multi-ethnic population of 10 million in North England. In our cohort of 2268 IMD patients, 1598 patients had general metabolic conditions (70.5%), and 670 had lysosomal storage disease/disorders (LSD)s (29.5%). The overall prevalence of ID and neurocognitive decline was found to be 15.7% (n = 357), with patients with LSDs accounting for 23.5% (n = 84) of affected patients. Given the prevalence of ID in adults with IMDs, access to multidisciplinary input from neuropsychology and neuropsychiatry services is important. Education of healthcare professionals to diagnose IMDs in patients with ID, in addition to neurocognitive and neuropsychiatric presentations, will avoid missed diagnoses of IMD and will have a positive effect on patient outcomes.

Acquisition and Development of Scrub Nursing Skills in Robotic-Assisted Cranial and Spinal Neurosurgical Procedures

Introduction. In high-tech operative specialties, including neurosurgery, the rise of interest in robotic systems is observed, since these techniques are hoped to increase the precision of the operation. However, for the optimal implementation of these solutions, the close cooperation between surgeon and perioperative nursing team as well as adequate coordination of their actions is required. These skills needs to be gained and trained. Aim. The following report describes the single center experience of the operating team in launching the robotic navigation in neurosurgical procedures from the vantage point of nursing team. Material and Methods. This work contains a phenomenological analysis of learning process and of optimalisation in cooperating the work of the involved operating team during early phase of introducing the robotic neurosurgical procedures (both cranial and spinal). Here, the typical problems during implementation of these techniques with separate analysis of single steps of preparing and applying the tool sets are described, where critical points and the potential of failure elimination is highlighted. Results. The preparing of the robotic surgical tool set is not demanding and differs only slightly from the preparation of standard sets designed for neuronavigation plus targeted cranial biopsy or percutaneous spine instrumentation, respectively. The key point is to synchronize the actions of the operating surgeon (-s), perioperative nurses and radiologic technicians. Conclusions. Implementation of robotic techniques is a challenge for the whole neurosurgical team, including the scrub/circulating nurses. What is more important in the initial phase than (usually intuitive) operation of the devices, is the coordination of single team members’ actions and developing the communication skills regarding the planned course of the surgery and potential modification of its course, needed before the surgery starts. (JNNN 2024;13(1):29–35)

Ambulatory bariatric surgery: a prospective single-center experience.

Ambulatory bariatric surgery has recently gained interest especially as a potential way to improve access for eligible patients with severe obesity. Building on our previously published research, this follow-up study delves deeper in the evolving landscape of ambulatory bariatric surgery over a 3-year period, focusing on predictors of success/failure. In a prospective single-center follow-up study, we conducted a descriptive assessment of all eligible patients as per our established protocol, who underwent a planned same-day discharge (SDD) primary sleeve gastrectomy (SG) or Roux-en-Y gastric bypass (RYGB) between 03/01/2021 and 02/29/2024. Trends in SDD surgeries over time were assessed over six discrete 6month intervals. Primary endpoint was defined as a successful discharge on the day of surgery without emergency department visit or readmission within 24h. Secondary outcomes included 30-day postoperative morbidity. A total of 811 primary SG and 325 RYGB procedures were performed during the study period. Among them, 30% (n = 244) were SDD-SGs and 6% (n = 21) were SDD-RYGBs, respectively. At baseline, median age of the entire SDD cohort was 43years old, 81% were females, and body mass index (BMI) was 44.5kg/m2. The planned SDD approach was successful in 89% after SG (n = 218/244) and in 90% after RYGB (n = 19/21). Nausea/vomiting was the main reason for a failed SDD approach after SG (46%). The 30-day readmission rate was 1.5% (n = 4) for the entire SDD cohort including only one readmission in the first 24h. The percentage of SDD-SGs performed as a proportion of total SGs increased over the initial five consecutive six-month intervals (14%, 25%, 24%, 38%, and 49%). Our SDD protocol for bariatric surgery demonstrates a favorable safety profile, marked by high success rate and low postoperative morbidity. These outcomes have led to a continued increase in ambulatory procedures performed over time especially SG.

Predictors of Intrahospital Mortality in Aneurysmal Subarachnoid Hemorrhage after Endovascular Embolization

Background and Objectives: Aneurysmal subarachnoid hemorrhage (ASAH) is defined as bleeding in the subarachnoid space caused by the rupture of a cerebral aneurysm. About 11% of people who develop ASAH die before receiving medical treatment, and 40% of patients die within four weeks of being admitted to hospital. There are limited data on single-center experiences analyzing intrahospital mortality in ASAH patients treated with an endovascular approach. Given that, we wanted to share our experience and explore the risk factors that influence intrahospital mortality in patients with ruptured intracranial aneurysms treated with endovascular coil embolization. Materials and Methods: Our study was designed as a clinical, observational, retrospective cross-sectional study. It was performed at the Department for Radiology, University Clinical Center Kragujevac in Kragujevac, Serbia. The study inclusion criteria were ≥18 years, admitted within 24 h of symptoms onset, acute SAH diagnosed on CT, aneurysm on DSA, and treated by endovascular coil embolization from January 2014 to December 2018 at our institution. Results: A total of 66 patients were included in the study—48 (72.7%) women and 18 (27.3%) men, and 19.7% of the patients died during hospitalization. After adjustment, the following factors were associated with in-hospital mortality: a delayed ischemic neurological deficit, the presence of blood in the fourth cerebral ventricle, and an elevated urea value after endovascular intervention, increasing the chances of mortality by 16.3, 12, and 12.6 times. Conclusions: Delayed cerebral ischemia and intraventricular hemorrhage on initial head CT scan are strong predictors of intrahospital mortality in ASAH patients. Also, it is important to monitor kidney function and urea levels in ASAH patients, considering that elevated urea values after endovascular aneurysm embolization have been shown to be a significant risk factor for intrahospital mortality.

Surgical strategy in treatment of metopic synostosis in a single centre experience: technical note and quantitative analysis of the outcomes.

Trigonocephaly is the most common craniosynostosis involving orbits. Although some degree of agreement has been reached regarding surgical timing and indications for treatment, there is no consensus regarding the ideal operative technique to guarantee an optimal morphological outcome. The purpose of this study is to describe both strategies and to compare morphological outcomes by means of morphological surface analysis obtained from three-dimensional (3D) stereophotogrammetry, with two different techniques. We retrospectively investigated 43 patients with metopic synostosis surgically treated between 2004 and 2020. Two different techniques were applied, addressed as technique A and B. Ten patients undergone postoperative 3d stereophotogrammetry were enrolled, and cephalometric measurements were taken and compared to a cohort of unaffected patients matched by age and gender. Comparison of the groups demonstrated a hypercorrection of the metopic angle of the second technique, associated with a slightly lower correction of the interfrontoparietal diameter. The metopic angle showed to be significantly undercorrected with the first method. Alternated barrel staving technique appears to be a quick and satisfactory method in cranial remodelling for metopic synostosis. It guarantees an optimal aesthetic result in the first years after surgery.

Genetic etiology in patients diagnosed with congenital hypothyroidism with new-generation sequencing: A single-center experience

AimCongenital hypothyroidism (CH) is the most common endocrine disorder of the newborn; it is seen in every 3000–4000 births. Genetic features can guide treatment for patients with in situ glands. The present study aimed to contribute to the literature on CH variants and to show the benefit that genetic analysis can provide to patients in follow-up. MethodA total of 52 patients (47 families) diagnosed with CH were included in the study. Overall, 32 target genes involved in thyroid physiology were investigated by next-generation sequencing (NGS). ResultsIn total, 29 (55 %) of the patients were male, and the rate of dysgenesis was 19.2 %. In this study, 29 of 52 patients had at least one variant in one gene involved in CH (n = 29, 33 different variants) (Including likely benign variants and variants of unknown significance). There were 21 patients (40.3 %) with gland in situ. The most common variant was DUOX2 (20 %). The second most common variants were those in the TPO and TG genes (15 % and 15 %, respectively); 41.1 % of these were variants of uncertain significance (VUS), 26.4 % were pathogenic, 23.5 % were likely benign, and 11.7 % were likely pathogenic. On the basis of their zygosity, we identified 73.5 % heterozygous, 17.6 % homozygous, and 8.9 % combined heterozygous variants. There were mutant variants in two genes in six patients and three in one patient. ConclusionThis study found a variant in 55 % of the patients and shed light on the etiology of some cases of CH. The frequency of VUS was high. Although variants were identified in this study, their implication in the etiology of CH is not certain and, for most of the patients, it is also not sufficient for explaining the pathology with the current state of knowledge.

The use of fosfomycin in infections caused by multidrug-resistant pathogens, especially pneumonia in children: a five-year retrospective single-centre experience.

Fosfomycin is gaining increasing attention for its activity against MDR or XDR pathogens. Currently, IV fosfomycin is a potential option for treating various infections, including urinary tract infections, pneumonia and skin infections when first-line treatments fail. To evaluate the demographic, clinical, microbiological and treatment modality of children received IV fosfomycin to treat infections caused by MDR pathogens since there are few data on the use of fosfomycin in children. This study was conducted retrospectively with patients under 18 years of age who were treated with IV fosfomycin for at least 72 h due to infections caused by MDR pathogens between January 2019 and October 2023 at Marmara University Pendik Training and Research Hospital, İstanbul, Türkiye. Data on demographic and clinical features, microbiological findings, treatment modalities and side effects were evaluated. Twenty-five children, for a total of 32 cases of infection episodes, with a mean age of 11.4 ± 3.92 years who received IV fosfomycin were included. The most frequent comorbidity was chronic pulmonary diseases, and the most common infection needed for IV fosfomycin was MDR Pseudomonas aeruginosa pneumonia. In all cases, fosfomycin was administered in combination with other antibiotics, mainly meropenem-colistin (68.7%) or meropenem (15.6%). Twenty-two (71.9%) cases had favourable clinical responses at the end of therapy. Our results suggest that IV fosfomycin may be an effective treatment option for MDR pathogens in the paediatric population. Nevertheless, careful stewardship is necessary to maintain efficacy and reduce antimicrobial resistance selection risk.