scholarly journals Determinants of Complicated Pneumonia in Hospitalized Pediatric Patients

2021 ◽  
Author(s):  
Mohammadreza Mirkarimi ◽  
Mohsen Alisamir ◽  
Parastoo Nasiri ◽  
Shahriar Barouti ◽  
Shooka Mohammadi
Keyword(s):  
Clinical Characteristics ◽  
Pediatric Patients ◽  
Length Of Hospital Stay ◽  
Failure To Thrive ◽  
Underlying Disease ◽  
Hospitalized Children ◽  
History Of ◽  
The Mean ◽  
Iranian Children ◽  
Complicated Pneumonia

Pediatric complicated pneumonia (PCOMP) is the leading cause of mortality in children under the age of five. The study was conducted to determine the epidemiological and clinical characteristics of children with PCOMP. A retrospective study was carried out among all pediatric patients who were hospitalized due to complicated pneumonia in Abuzar Hospital (Ahvaz, Iran) during two years. The patients were evaluated in terms of epidemiological and clinical characteristics. A total of 65 hospitalized children and infants were identified. More than half of the patients were females (n=36; 55.3%). Their mean age was 4.21±3.80 years (range six months-15 years), and 64.1% of them (n=42) were under the age of five. There were 12 (19.4%) patients with failure to thrive (FTT). In addition, 58.5% of patients (n=38) had no history of hospitalization, and 66.2% of them (n=43) did not have any underlying disease. The mean length of hospital stay (LOS) was 12.46±6.85 (range 4-45) days. Admission was more common in winter (40%) and autumn (33.8%). Moreover, there were no significant associations between the types of complications and patients’ gender, age, FTT, and LOS. Further studies are warranted to identify factors contributing to disease severity and develop appropriate strategies for the prevention and treatment of PCOMP among Iranian children.

scholarly journals 1339. Antibiotic Prescribing Varies among Patients with a Documented Penicillin or Cephalosporin Adverse Drug Reaction

2020 ◽  
Vol 7 (Supplement_1) ◽  
pp. S681-S681
Author(s):  
Brian R Lee ◽  
Jason Newland ◽  
Jennifer Goldman
Keyword(s):  
Pediatric Patients ◽  
Antibiotic Prescribing ◽  
Hospitalized Children ◽  
Beta Lactam ◽  
Research Support ◽  
Point Prevalence Study ◽  
History Of ◽  
Gi Infections ◽  
To Receive ◽  
Indication For Treatment

Abstract Background Studies have shown that over half of hospitalized children receive an antibiotic during their encounter, of which between 30-50% is considered inappropriate. Antibiotic prescribing is further complicated as approximately 10% of children are labeled beta-lactam allergic, resulting in the use of either broad-spectrum or suboptimal therapy. The purpose of this study was to compare antibiotic prescribing between patients with a documented ADR vs. those without using a nationwide sample of hospitalized children. Methods We performed a point prevalence study among 32 hospitals between July 2016-December 2017 where data were collected via chart review on pediatric patient and antimicrobial characteristics, including the indication for all antimicrobials. In additional, ADR history data were collected on which antimicrobial(s) were documented (e.g., penicillin, cephalosporins). Patients were mutually assigned into either: 1) no documented ADR; 2) penicillin ADR-only; 3) cephalosporin ADR-only; and 4) ADR for both penicillin and cephalosporin. The distribution of antibiotics were compared between the ADR groups, stratified by the indication for treatment. Results A total of 12,250 pediatric patients (17,929 antibiotic orders) who were actively receiving antibiotics were identified. A history of penicillin and cephalosporin ADR was documented in 5.5% and 2.8% of these patients, respectively. When compared to patients with no documented ADR, penicillin ADR patients were more likely to receive a fluoroquinolone for a SSTI infection (odds ratio [OR]: 5.6), surgical prophylaxis (OR: 18.8) or for surgical treatment (OR: 5.2) (see Figure). Conversely, penicillin ADR patients were less likely to receive first-line agents, such as narrow-spectrum penicillin for bacterial LRTI (OR: 0.08) and piperacillin/tazobactam for GI infections (OR: 0.22). Cephalosporin ADR patients exhibited similar patterns with increased use of carbapenems and fluoroquinolones when compared to patients with no ADR. Figure 1: Odds of Receiving Select Antimicrobials Among PCN ADR Patients When Compared to Non-ADR patients, by Indication Conclusion A large, nationwide sample of pediatric patients who were actively prescribed antibiotics helped identify several diagnoses where comprehensive guidelines for appropriate ADR prescribing and increased ADR de-labeling initiatives are needed to ensure optimal treatment. Disclosures Brian R. Lee, MPH, PhD, Merck (Grant/Research Support) Jason Newland, MD, MEd, FPIDS, Merck (Grant/Research Support)Pfizer (Other Financial or Material Support, Industry funded clinical trial)

Feasibility of Powered Intracapsular Tonsillectomy in Pediatric Patients with Tonsil Problem, Including Recurrent Tonsillitis: A Single Surgeon’s Experience

ORL ◽  
2020 ◽  
Vol 82 (6) ◽  
pp. 335-342
Author(s):  
Heejin Kim ◽  
Sung Won Li ◽  
Sung Min Park ◽  
Sung Kyun Kim ◽  
Seok Jin Hong ◽  
...  
Keyword(s):  
Obstructive Sleep Apnea ◽  
Sleep Apnea ◽  
Postoperative Pain ◽  
Pediatric Patients ◽  
Recurrent Tonsillitis ◽  
Obstructive Sleep ◽  
History Of ◽  
The Mean ◽  
Intracapsular Tonsillectomy

<b><i>Introduction:</i></b> Powered intracapsular tonsillectomy (PIT) is a technique that protects the tonsillar capsule by using a microdebrider, resulting in faster wound-healing and reduced suffering. Many studies have found PIT to be effective, particularly in pediatric patients with obstructive sleep apnea (OSA). However, previous studies have not included patients with a history of recurrent tonsillitis. <b><i>Objective:</i></b> The aim of this study was to determine the efficacy of PIT in pediatric patients even with a history of recurrent tonsillitis, and therefore, we want to expand the indication for PIT and reveal its safety. <b><i>Methods:</i></b> A total of 886 pediatric patients underwent PIT between February 2013 and March 2016. All patients rated their postoperative pain using a visual analog scale (VAS) and completed the Korean obstructive sleep apnea (KOSA)-18 questionnaire for assessment of their quality of life (QOL). There were 539 males and 347 females. Their mean age was 6.2 years (range 2–14 years). The majority (77.7%) underwent the operation for OSA, and the rest (22.3%) had a history of recurrent tonsillitis. To compare the efficacy of PIT with traditional tonsillectomy, we selected 191 patients who underwent extracapsular tonsillectomy (ECT), a conventional technique, during the same time period. The median follow-up period was 16.7 months. During the follow-up period, instances of delayed bleeding and recurrent pharyngitis were monitored. <b><i>Results:</i></b> In comparison to the patients who underwent ECT, the PIT group showed significantly fewer cases of postoperative bleeding (<i>p</i> = 0.027). Thirteen patients in the PIT group (1.5%) visited the hospital during the follow-up period for pharyngitis, while 8 in the ECT group (4.2%) visited for pharyngitis. The mean postoperative pain score, as assessed by a VAS, was 4.6 ± 3.2, and pain improved within an average of 2.9 days after surgery in the PIT group. The mean KOSA-18 score for the QOL of the patients was 65.9 preoperatively and 35.6 postoperatively in the PIT group. <b><i>Conclusions:</i></b> Pediatric tonsillectomy using PIT is valid for reducing postoperative pain and improving the QOL of OSA patients. PIT is also effective and safe for patients with a history of recurrent tonsillitis.

Intra-Abdominal Venous Thrombosis: Characteristics of Pediatric and Adult Patients

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4219-4219
Author(s):  
Daniel Landi ◽  
Michele Beckman ◽  
Nirmish Shah ◽  
Paula Bockenstedt ◽  
John A. Heit ◽  
...  
Keyword(s):  
Lower Extremity ◽  
Research Funding ◽  
Clinical Characteristics ◽  
Pediatric Patients ◽  
Adult Patients ◽  
Renal Veins ◽  
Vein Thrombosis ◽  
The Mean ◽  
Hepatic Portal ◽  
Morbid Conditions

Abstract Abstract 4219 Background: Clinical characteristics associated with abdominal vein thrombosis (including hepatic, portal, and mesenteric veins; renal veins; and inferior vena cava [IVC]) overlap with characteristics associated with deep vein thrombosis (DVT) in the legs and pulmonary embolism (PE) but also possess unique attributes. These characteristics may differ between adult and pediatric patient groups. Methods: Using a standardized data-collection form, demographic and baseline characteristics were prospectively collected from consecutive consenting patients enrolled within one of seven Thrombosis and Hemostasis Centers over a seven-year period, August 2003 to June 2010. Patients with intra-abdominal venous thrombosis (defined as abdominal and renal veins, and IVC) were divided into pediatric (age <18 years) and adult (age ≥18 years) groups and compared to patients in the same age ranges with lower extremity DVT and/or PE. RESULTS: As of June 2010, the Patient Registry contained a total of 3105 patients with venous thromboembolism (VTE). A total of 270 patients had intra-abdominal thrombosis (9%) compared to 2276 with lower extremity DVT and/or PE (73%). Of the intra-abdominal clots, pediatric patients were more likely to have IVC thrombus (22 of 42; 52%) than adults (38 of 228; 17%; p<0.0001); conversely, pediatric patients were less likely to have hepatic, portal, and mesenteric thrombosis (n=25; 60%) than adults (n=195; 86%, p<.0001). For adult patients, proportionately more women had abdominal thrombosis (157 of 228; 68.9%) than DVT/PE (1306 of 2140; 61%; p=0.02); in contrast, comparable numbers of female pediatric patients had abdominal thrombosis (20 of 42; 47.6%) and DVT/PE (68 of 136; 50%). The mean age for patients having abdominal thrombosis was lower than patients having DVT/PE for both adults (38.3 vs 43.0 yrs; p<0.0001) and pediatric patients (9.0 vs 11.9 yrs; p=0.014). Similarly, the mean body mass index was lower for patients having abdominal thrombosis than patients having DVT/PE for both adults (28.1 vs 30.8; p<0.0001) and pediatric patients (21.5 vs. 24.7; p=0.05). There were no relationships with race, ethnicity, or inherited thrombophilic disorders. In adults, autoimmune conditions (21.9 vs 13.6; p=0.0007) and recent surgery (14.0 vs 9.2; p=0.02) were more common in patients with abdominal thrombosis, whereas, hyperlipidemia was more frequent in patients with DVT/PE (8.3 vs 13.2; p=0.04). None of these relationships were seen in the pediatric patients, but trauma was more frequently seen in pediatric patients with DVT/PE than abdominal thrombosis. Sixty-one adult (26.8%) and 6 pediatric patients (14.3%) had another VTE elsewhere, with most occurring after the initial event (21 DVT and 7 PE). Similar proportions of adult and pediatric patients had arterial thromboembolic events for both abdominal thrombosis and DVT/PE. CONCLUSIONS: Adult patients with abdominal thrombosis are more likely female, have hepatic, portal and/or mesenteric vein thrombosis, and common co-morbid conditions include recent surgery and autoimmune disorders. Pediatric patients are evenly divided between male and female, more commonly have IVC thrombosis, and co-morbid conditions include recent trauma. Both adult and pediatric patients with abdominal thrombosis tend to be younger and have a lower BMI than patients with DVT and/or PE. We also found a high rate of concurrent thrombosis in both adult and pediatric patients with abdominal thrombosis. In summary, patients with abdominal thrombosis display different clinical characteristics when compared to those with DVT/PE, specifically in terms of gender, location of thrombosis, and associated co-morbidities. Some of these differences, such as higher rates of IVC thrombosis in pediatric patients with abdominal thrombosis, might be caused by variance between genetic and anatomic factors. Further studies are warranted to explain these differences. Disclosures: Kulkarni: Novo Nordisk: Membership on an entity's Board of Directors or advisory committees, Participate in clinical trials. Philipp:Baxter: Research Funding; Wyeth: Research Funding; Octapharma: Research Funding. Ortel:Sanofi-Aventis: Consultancy, Research Funding; Ortho-McNeil: Consultancy; GlaxoSmithKline: Research Funding; Eisai: Research Funding; Pfizer: Research Funding.

Carbohydrate metabolism in patients with Cushing disease: a glance at the incretin system

2016 ◽  
Vol 62 (5) ◽  
pp. 67-68
Author(s):  
Lubov V. Matchekhina ◽  
Ekaterina A. Shestakova ◽  
Zhanna E. Belaya ◽  
Marina V. Shestakova
Keyword(s):  
Drug Therapy ◽  
Carbohydrate Metabolism ◽  
Previous History ◽  
Pituitary Surgery ◽  
Underlying Disease ◽  
Control Group ◽  
Cushing Disease ◽  
Free Cortisol ◽  
History Of ◽  
The Mean

Introduction. The relevance of carbohydrate metabolism studying in patients with Cushing disease can be explained by frequent occurrence of glucose metabolism disturbances on the one hand, and difficulties in glucose-lowering therapy in these patients on the other. The effectiveness of hyperglycaemia treatment may be reduced due to difficulties in remission / cure of the underlying disease, as well as to the use of specific drug-therapy, leading to the hyperglycaemia. There is a growing interest in research aimed at studying the role of incretin system in the pathogenesis of secondary hyperglycemia associated with neuroendocrine diseases recently.Methods. A total of 20 patients with Cushing disease were included, (19 female and 1 male), the mean age was 37.5 years (18-69). All of the patients were diagnosed with Cushing disease for the first time (using urinary free cortisol levels and MRI-data); none of them had a history of previous drug therapy, radiotherapy or pituitary surgery. The mean HbA1c level was 5,8% (5,3-6,2). All patients underwent OGTT, during which glucose, glucagon, GLP1, GLP2, GIP, ghrelin were measured at 0, 30 and 120 min respectively. The control group included 21 patients without previous history of carbohydrate metabolism disturbances. After OGTT 57% were presented without any carbohydrate metabolism disturbances, 28,57% presented with prediabetes and 14,43% were diagnosed with diabetes.Results. After glucose levels analyzing 40% of patients were diagnosed with early carbohydrate metabolism disturbances ,15% were diagnosed with diabetes. After glucose intake a slight inrease in glucagon levels with a peak by 30’ (p=0,001) compared to gradually decreasing levels in controls was observed . The levels of GIP during OGTT were not significantly different compared to control group. GLP-1 and GLP-2 levels were significantly higher compared to controls (p=0,017 and p<0,001 respectively) with peak levels at 30’. Ghrelin levels were also significantly higher compared to controls (p=0,013)Conclusion. Incretins levels can be possible markers of specific carbohydrate metabolism disturbances in patients with Cushing disease and presumably will help to differentiate steroid diabetes from T2DM. Further investigations needed to prove these speculations.

scholarly journals A study on epidemiological and clinical characteristics of measles patients admitted to Sir Ronald Ross institute of tropical and communicable diseases, Hyderabad, India

2019 ◽  
Vol 6 (3) ◽  
pp. 1345
Author(s):  
Asma . ◽  
Anupama Pulla ◽  
Vimala Thomas
Keyword(s):  
Clinical Characteristics ◽  
Immunization Coverage ◽  
Communicable Diseases ◽  
Childhood Mortality ◽  
Cross Sectional ◽  
Measles Outbreak ◽  
History Of ◽  
Study Population ◽  
The Mean ◽  
Disease Hospital

Background: Measles is an acute infectious exanthematous disease of childhood caused by paramyxovirus. With increase in immunization coverage levels, the intensity of measles outbreak has decreased in the country. In some parts of India, measles is still a major cause of morbidity and childhood mortality. Objective of the study was to determine the epidemiological and clinical characteristics of patients with macular/maculo-papular rash.Methods: A cross sectional record based retrospective study was carried out at Sir Ronald Ross institute of tropical and communicable diseases, Hyderabad. All the cases admitted in year 2016 with history of fever and macular/maculo-papular rash were included in the study. The study was carried out for 2 months.Results: A total of 639 cases were admitted to infectious disease hospital during the year 2016 with history of fever and rash. The mean age of study population was 6.08±4.9 years. Around 43.7% of cases were in the age group of 1–5 years. The disease was slightly higher among boys (52.3%) as compared to girls (47.7%). Amongst the clinical features, fever and rash were seen in 100%, coryza in 80%, and conjunctivitis in 63.3% of study population.Conclusions: Majority of the children suffering from measles was not vaccinated and the most common reason for failure to immunize children was lack of awareness. There is a need to increase awareness about importance of measles immunization to reduce under five mortality.

scholarly journals Association of Underlying Diseases and Clinical Characteristics with Mortality in Patients with 2019 Novel Coronavirus in Iran

2020 ◽  
Vol 15 (5) ◽  
Author(s):  
Mohammadhossein Zamanian ◽  
Zohre Foroozanfar ◽  
Zhila Izadi ◽  
Samira Jafari ◽  
Hossein Derakhshankhah ◽  
...  
Keyword(s):  
Heart Disease ◽  
Clinical Characteristics ◽  
Underlying Disease ◽  
Test Results ◽  
Rt Pcr ◽  
Factors Associated ◽  
History Of ◽  
Novel Coronavirus ◽  
Underlying Diseases ◽  
Pcr Test

Objectives: The first case of 2019 novel coronavirus disease (COVID-19) was reported in Iran in February 2020. Here, we report the epidemiological and clinical characteristics of patients with COVID-19 and factors associated with mortality in these patients. Methods: A retrospective cohort study was conducted from February 22, 2020, to March 24, 2020, in Golestan Hospital in Kermanshah, Iran. Demographic data including underlying diseases and clinical data including the presenting symptoms, chest computed tomography (CT) scan, reverse transcription polymerase chain reaction (RT-PCR) test results, and outcomes were extracted from electronic medical records. Simple and multiple logistic regression methods were used to explore the factors associated with mortality. Results: Of 245 patients admitted with COVID-19, 155 (63.30%) were male. The mean age of the subjects was 54.68 ± 19.21. Forty-five (18.48%) patients had underlying diseases. Common symptoms were dyspnea (n = 137; 55.9%), cough (n = 93; 38.0%), and fever (n = 78; 31.8%). All patients had pneumonia with abnormal findings on chest CT scan (100%), and RT-PCR test results were positive in 87 (35.50%) patients. Of the total admitted cases, 38 (15.5%) patients died during hospitalization. An old age (OR = 1.09; 95% CI: 1.02 to 1.06), history of heart disease (OR = 5.07; 95% CI: 1.46 to 17.58), hypertension (OR = 5.82; 95% CI: 1.13 to 30.04), smoking (OR = 11.44; 95% CI: 1.01 to 29.53), history of at least one underlying disease (OR = 3.31; 95%CI: 1.54 to 7.09), and symptoms of decreased consciousness at the time of admission (OR = 24.23; 95% CI: 2.62 to 223.39) were associated with mortality. Also, the symptoms of cough (OR = 0.383; 95% CI: 0.17 to 0.88) and fever (OR = 0.278; 95% CI: 0.10 to 0.74) had a negative association with mortality. Conclusions: In the current study, factors including old age, smoking, symptoms of decreased consciousness, and underlying diseases such as heart disease, hypertension, and history of at least one underlying disease were associated with mortality. Factors associated with mortality should be considered so that we can better manage patients with COVID-19.

scholarly journals Evaluating bronchodilator response in pediatric patients with post-infectious bronchiolitis obliterans: use of different criteria for identifying airway reversibility

2016 ◽  
Vol 42 (3) ◽  
pp. 174-178 ◽  
Author(s):  
Rita Mattiello ◽  
Paula Cristina Vidal ◽  
Edgar Enrique Sarria ◽  
Paulo Márcio Pitrez ◽  
Renato Tetelbom Stein ◽  
...  
Keyword(s):  
Airway Obstruction ◽  
Bronchiolitis Obliterans ◽  
Pediatric Patients ◽  
Bronchodilator Response ◽  
Peripheral Airway ◽  
Pediatric Pulmonology ◽  
History Of ◽  
Asthma And Allergy ◽  
The Mean ◽  
Post Infectious

ABSTRACT Objective: Post-infectious bronchiolitis obliterans (PIBO) is a clinical entity that has been classified as constrictive, fixed obstruction of the lumen by fibrotic tissue. However, recent studies using impulse oscillometry have reported bronchodilator responses in PIBO patients. The objective of this study was to evaluate bronchodilator responses in pediatric PIBO patients, comparing different criteria to define the response. Methods: We evaluated pediatric patients diagnosed with PIBO and treated at one of two pediatric pulmonology outpatient clinics in the city of Porto Alegre, Brazil. Spirometric parameters were measured in accordance with international recommendations. Results: We included a total of 72 pediatric PIBO patients. The mean pre- and post-bronchodilator values were clearly lower than the reference values for all parameters, especially FEF25-75%. There were post-bronchodilator improvements. When measured as mean percent increases, FEV1 and FEF25-75%, improved by 11% and 20%, respectively. However, when the absolute values were calculated, the mean FEV1 and FEF25-75% both increased by only 0.1 L. We found that age at viral aggression, a family history of asthma, and allergy had no significant effects on bronchodilator responses. Conclusions: Pediatric patients with PIBO have peripheral airway obstruction that is responsive to treatment but is not completely reversible with a bronchodilator. The concept of PIBO as fixed, irreversible obstruction does not seem to apply to this population. Our data suggest that airway obstruction is variable in PIBO patients, a finding that could have major clinical implications.

scholarly journals Characteristics and long-term outcomes of childhood glaucoma: a retrospective-cohort study

F1000Research ◽  
2021 ◽  
Vol 10 ◽  
pp. 165
Author(s):  
Supawan Surukrattanaskul ◽  
Pukkapol Suvannachart ◽  
Sunee Chansangpetch ◽  
Anita Manassakorn ◽  
Visanee Tantisevi ◽  
...  
Keyword(s):  
Clinical Characteristics ◽  
Secondary Glaucoma ◽  
Research Network ◽  
Congenital Glaucoma ◽  
Long Term Outcomes ◽  
History Of ◽  
The Mean ◽  
Primary Glaucoma

Purpose: To evaluate the clinical characteristics and treatment outcomes of patients with childhood glaucoma. Methods: We retrospectively reviewed the data of patients with childhood glaucoma who visited the glaucoma clinics at the Queen Sirikit National Institute of Child Health and the King Chulalongkorn Memorial Hospital between January 2008 and January 2018. The diagnosis was based on the Childhood Glaucoma Research Network classification. We recorded their clinical characteristics and requirement of any glaucoma interventions. Results: A total of 691 eyes from 423 patients were included in this study. The patients predominantly comprised boys. The average follow-up duration was 71.3±63.8 months. The mean age at presentation was 3.9±4.4 years. Most patients presented with a high initial intraocular pressure (IOP) of 28.5±11.2 mmHg. Glaucoma associated with non-acquired ocular anomalies (22.9%) was the most common subtype, followed by primary congenital glaucoma (20.8%). We recorded a family history of glaucoma in 6.4% of patients. Most patients had bilateral glaucoma (63.4%) and required at least one intervention (51.5%). The average IOP at the latest follow-up visit was 19.1±10.8 mmHg. All glaucoma types had significantly lower IOP, compared to that at their baselines (all p<0.001). Moreover, most patients had an unfavourable visual acuity (49.5%) at their latest visit. Conclusions: Secondary glaucoma associated with non-acquired ocular anomalies is the most common subtype of glaucoma. All subtypes, including primary glaucoma, were sporadic. The majority of patients had unfavourable visual outcomes. These real-world findings are fundamental to acquire a better understanding of childhood glaucoma.

scholarly journals Different Clinical Characteristics of Paroxysmal Nocturnal Hemoglobinuria in Pediatric and Adult Patients

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3341-3341
Author(s):  
Alvaro Urbano-Ispizua ◽  
Petra Muus ◽  
Hubert Schrezenmeier ◽  
Antonio Medina Almeida ◽  
Amanda Wilson ◽  
...  
Keyword(s):  
Research Funding ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Clinical Characteristics ◽  
Pediatric Patients ◽  
Adult Patients ◽  
Vascular Event ◽  
Clone Size ◽  
History Of ◽  
Pnh Clone ◽  
Hypoplastic Anemia

Abstract Introduction: Studies of children with paroxysmal nocturnal hemoglobinuria (PNH) are scarce and include a very limited number of patients. The objective of this analysis was to describe characteristics of PNH at enrollment for the largest available registry of pediatric patients, and to compare demographic and clinical characteristics with those of adult patients. Methods: The International PNH Registry is a prospective, multi-center worldwide, observational study of patients with a PNH clone of 0.01-100%. Data are collected from patient medical records at the time of Registry enrollment and every six months thereafter. Adult patients were ³18 years of age at enrollment and disease start and pediatric patients were <18 years at enrollment. Demographics and clinical parameters in patients untreated with eculizumab at enrollment for the two age cohorts were compared using the Wilcoxon-Mann-Whitney test for medians and PearsonÕs chi-square for frequencies. The rate of thrombotic events (TE) between disease start (defined as the earliest reported PNH symptoms, granulocyte clone, or PNH diagnosis) and enrollment was calculated per 100 person-years. Results: As of March 2, 2015, a total of 2,184 patients were eligible for analysis: 94 (4.3%) pediatric patients and 2,090 (95.7%) adult patients. Median age (range) at enrollment was 14.0 years (3-17) in pediatrics and 45.5 years (18-100) in adults; median disease duration was 0.7 years and 2.1 years, respectively (p<0.001). More pediatric than adult patients had a PNH clone of <10% and severe cytopenia (Table). Pediatric patients had lower percent of reticulocytes compared with adults (2.1% vs. 2.6%, respectively; p=0.015). History of aplastic or hypoplastic anemia was more frequent in pediatric than adult patients (76.5% vs 54.4%, respectively; p<0.001). History of TE and any major adverse vascular event was less frequent in pediatrics (2.1% vs 8.7%; p=0.025, and 4.3% vs. 14.4%; p=0.005). The rate of TE between disease start and enrollment was lower in pediatric patients, but not statistically significant: 1.4 per 100 person-years (95%CI 0.2-5.2) compared to adult patients (2.3 per 100 person-years (95%CI 2.0-2.6). More pediatric patients than adults had abdominal pain at enrollment. Conclusions: The International PNH Registry provides the largest available pediatric cohort of patients with a PNH clone to characterize this understudied population and demonstrate an important disease burden. Pediatric patients were more likely to have smaller PNH clones and a higher component of aplastic/hypoplastic anemia. Pediatric patients had fewer vascular events. These findings may reflect the natural evolution of the disease and can be useful in the clinical management of PNH. Table 1. Clinical Characteristics at Enrollment of Pediatric and Adult Patients with PNH Pediatric(n=94) Adult(n=2,090) P-value Clone size (percent GPI-deficient granulocytes), n (%)<10% 10 to < 50% ³50% 47 (55.3) 16 (18.8) 22 (25.9) 550 (38.3) 322 (22.4) 565 (39.3) 0.006* Cytopenia status, n (%)None (neutrophils ³ 1.5 x 109/L and platelets ³100 x 109/L) Moderate (neutrophils <1.5 x 109/L or platelets <100 x 109/L) Severe (neutrophils <0.5 x 109/L or platelets <20x109/L) 22 (29.3) 28 (37.3) 25 (33.3) 735 (42.2) 784 (45.1) 221 (12.7) <0.001* Percent reticulocytesMedian (Q1, Q3) 2.1 (1.1, 3.5) 2.6 (1.6, 4.6) 0.015 Hemolytic status, n (%)Hemolytic (LDH ³1.5 x ULN and/or reticulocytes ³60 x 109/L) Not hemolytic (LDH <1.5 x ULN and reticulocytes <60 x 109/L) 33 (58.9) 23 (41.1) 1,038 (65.3) 551 (34.7) NS LDH Ratio, n (%)<1.5 x ULN³1.5 x ULN 30 (58.8) 21 (41.2) 684 (47.0) 770 (53.0) NS History of TE, n (%)Yes No 2 (2.1) 92 (97.9) 181 (8.7) 1,902 (91.3) 0.025 Rate of TENumber of TE, n Person-years (disease start to enrollment) Rate/100 person-years (95% CI) 2 139.9 1.4 (0.2-5.2) 255 11,119.8 2.3 (2.0-2.6) NS History of MAVE, n (%) 4 (4.3) 300 (14.4) 0.005 GPI, glycosylphosphatidylinositol; LDH, lactate dehydrogenase; MAVE, major adverse vascular event; TE, thrombotic event; ULN, upper limit of normal *P-values for clone size and cytopenia status represent overall comparison of categories. Disclosures Muus: Alexion Pharmaceuticals: Honoraria. Schrezenmeier:Alexion Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Almeida:Celgene: Consultancy; Novartis: Consultancy; Bristol Meyer Squibb: Speakers Bureau; Shire: Speakers Bureau. Wilson:Alexion Pharmaceuticals: Employment. Ware:Bayer Pharmaceuticals: Consultancy; Biomedomics: Research Funding; Eli Lilly: Other: DSMB membership; Bristol Myers Squibb: Research Funding.

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