Browsing by Subject "pediatrics"

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  • Dimitri, Paul; Fernandez-Luque, Luis; Banerjee, Indraneel; Bergada, Ignacio; Calliari, Luis Eduardo; Dahlgren, Jovanna; de Arriba, Antonio; Lapatto, Risto; Reinehr, Thomas; Senniappan, Senthil; Thomas-Teinturier, Cecile; Tsai, Meng-Che; Zaini, Azriyanti Anuar; Bagha, Merat; Koledova, Ekaterina (2021)
    Background: The use of technology to support health and health care has grown rapidly in the last decade across all ages and medical specialties. Newly developed eHealth tools are being implemented in long-term management of growth failure in children, a low prevalence pediatric endocrine disorder. Objective: Our objective was to create a framework that can guide future implementation and research on the use of eHealth tools to support patients with growth disorders who require growth hormone therapy. Methods: A total of 12 pediatric endocrinologists with experience in eHealth, from a wide geographical distribution, participated in a series of online discussions. We summarized the discussions of 3 workshops, conducted during 2020, on the use of eHealth in the management of growth disorders, which were structured to provide insights on existing challenges, opportunities, and solutions for the implementation of eHealth tools across the patient journey, from referral to the end of pediatric therapy. Results: A total of 815 responses were collected from 2 questionnaire-based activities covering referral and diagnosis of growth disorders, and subsequent growth hormone therapy stages of the patient pathway, relating to physicians, nurses, and patients, parents, or caregivers. We mapped the feedback from those discussions into a framework that we developed as a guide to integration of eHealth tools across the patient journey. Responses focused on improved clinical management, such as growth monitoring and automation of referral for early detection of growth disorders, which could trigger rapid evaluation and diagnosis. Patient support included the use of eHealth for enhanced patient and caregiver communication, better access to educational opportunities, and enhanced medical and psychological support during growth hormone therapy management. Given the potential availability of patient data from connected devices, artificial intelligence can be used to predict adherence and personalize patient support. Providing evidence to demonstrate the value and utility of eHealth tools will ensure that these tools are widely accepted, trusted, and used in clinical practice, but implementation issues (eg, adaptation to specific clinical settings) must be addressed. Conclusions: The use of eHealth in growth hormone therapy has major potential to improve the management of growth disorders along the patient journey. Combining objective clinical information and patient adherence data is vital in supporting decision-making and the development of new eHealth tools. Involvement of clinicians and patients in the process of integrating such technologies into clinical practice is essential for implementation and developing evidence that eHealth tools can provide value across the patient pathway.
  • Hintsala, Emilia (Helsingfors universitet, 2010)
    TAUSTA: Syöpään liittyvää angiogeneesiä eli verisuonten uudismuodostusta on tutkittu hyvin paljon mm. mahdollisena syöpähoitojen kohteena. Kiinteissä kasvaimissa ja leukemioissa on havaittu esimerkiksi angiogeneesiä säätelevien molekyylien tuotannon ja syöpäkudoksen ympäristön hiussuonitiheyden lisääntymistä. Tämän tutkimuksen tarkoituksena oli selvittää kahden angiogeneesiä säätelevän molekyylin, VEGF:n ja Angiopoietiini-2:n, pitoisuuksia lapsisyöpäpotilailla. METODIT: Määritimme VEGF- ja Angiopoietiini-2-pitoisuudet 49 kiinteäkasvainpotilaan plasmanäytteistä ja 35 leukemiapotilaan plasma- ja luuydinnäytteistä diagnoosivaiheessa ja hoidon aloittamisen jälkeen. TULOKSET: Kiinteäkasvainryhmässä mitattiin korkeampia VEGF-pitoisuuksia kuin leukemiaryhmässä ja pitoisuudet laskivat hoidon aloittamisen seurauksena. Leukemiaryhmässä mitattiin kiinteitä kasvaimia korkeampia Angiopoietiini-2-pitoisuuksia ja pitoisuudet laskivat hoidon aikana. Leukemiapotilailla hoidon aloittamisen jälkeen todettu korkea luuytimen VEGF-pitoisuus liittyi matalampaan elossaolo-osuuteen. VEGF- ja Angiopoietiini-2-pitoisuudet korreloivat plasma- ja luuydinnäytteiden välillä. Diagnoosivaiheessa lymfoomapotilailla oli korkeampia ja aivokasvainpotilailla matalampia Angiopoietiini-2-pitoisuuksia kuin muilla kiinteäkasvainpotilailla ja AML-potilailla korkeampia VEGF-pitoisuuksia kuin ALL-potilailla. PÄÄTELMÄT: Tutkimus antaa uutta tietoa lapsisyöpäpotilaiden angiogeneettisistä tekijöistä. VEGF- ja Angiopoietiini-2-pitoisuuksissa havaittiin eroja eri syöpätyyppien välillä ja muutoksia hoidon aloittamisen seurauksena. VEGF-pitoisuudella oli myös ennusteellista merkitystä.
  • Løhmann, Ditte J. A.; Asdahl, Peter H.; Abrahamsson, Jonas; Ha, Shau-Yin; Jónsson, Ólafur G.; Kaspers, Gertjan J. L.; Koskenvuo, Minna; Lausen, Birgitte; De Moerloose, Barbara; Palle, Josefine; Zeller, Bernward; Sung, Lillian; Hasle, Henrik (2019)
    Background Associations between body mass index (BMI), outcome, and leukemia-related factors in children with acute myeloid leukemia (AML) remain unclear. We investigated associations between pretherapeutic BMI, cytogenetic abnormalities, and outcome in a large multinational cohort of children with AML. Methods We included patients, age 2-17 years, diagnosed with de novo AML from the five Nordic countries (2004-2016), Hong Kong (2007-2016), the Netherlands and Belgium (2010-2016), and Canada and USA (1995-2012). BMI standard deviations score for age and sex was calculated and categorized according to the World Health Organization. Cumulative incidence functions, Kaplan-Meier estimator, Cox regression, and logistic regression were used to investigate associations. Results In total, 867 patients were included. The median age was 10 years (range 2-17 years). At diagnosis, 32 (4%) were underweight, 632 (73%) were healthy weight, 127 (15%) were overweight, and 76 (9%) were obese. There was no difference in relapse risk, treatment-related mortality or overall mortality across BMI groups. The frequency of t(8;21) and inv(16) increased with increasing BMI. For obese patients, the sex, age, and country adjusted odds ratio of having t(8;21) or inv(16) were 1.9 (95% confidence interval (CI) 1.1-3.4) and 2.8 (95% CI 1.3-5.8), respectively, compared to healthy weight patients. Conclusions This study did not confirm previous reports of associations between overweight and increased treatment-related or overall mortality in children. Obesity was associated with a higher frequency of t(8;21) and inv(16). AML cytogenetics appear to differ by BMI status.
  • Rautamo, Maria M; Kvarnström, Kirsi; Siven, Mia; Airaksinen, Marja; Lahdenne, Pekka Olavi; Sandler, Niklas (2020)
    The utilization of three-dimensional (3D) printing technologies as innovative manufacturing methods for drug products has recently gained growing interest. From a technological viewpoint, proof-of-concept on the performance of different printing methods already exist, followed by visions about future applications in hospital or community pharmacies. The main objective of this study was to investigate the perceptions of healthcare professionals in a tertiary university hospital about oral 3D-printed medicines for pediatric patients by means of focus group discussions. In general, the healthcare professionals considered many positive aspects and opportunities in 3D printing of pharmaceuticals. A precise dose as well as personalized doses and dosage forms were some of the advantages mentioned by the participants. Especially in cases of polypharmacy, incorporating several drug substances into one product to produce a polypill, personalized regarding both the combination of drug substances and the doses, would benefit drug treatments of several medical conditions and would improve adherence to medications. In addition to the positive aspects, concerns and prerequisites for the adoption of 3D printing technologies at hospital settings were also expressed. These perspectives are suggested by the authors to be focus points for future research on personalized 3D-printed drug products.
  • Bonamy, Anna-Karin Edstedt; Mohlkert, Lilly-Ann; Hallberg, Jenny; Liuba, Petru; Fellman, Vineta; Domellof, Magnus; Norman, Mikael (2017)
    Background-Advances in perinatal medicine have increased infant survival after very preterm birth. Although this progress is welcome, there is increasing concern that preterm birth is an emerging risk factor for hypertension at young age, with implications for the lifetime risk of cardiovascular disease. Methods and Results-We measured casual blood pressures (BPs) in a population-based cohort of 6-year-old survivors of extremely preterm birth (<27 gestational weeks; n=171) and in age-and sex-matched controls born at term (n=172). Measured BP did not differ, but sex, age-, and height-adjusted median z scores were 0.14 SD higher (P=0.02) for systolic BP and 0.10 SD higher (P=0.01) for diastolic BP in children born extremely preterm than in controls. Among children born extremely preterm, shorter gestation, higher body mass index, and higher heart rate at follow-up were all independently associated with higher BP at 6 years of age, whereas preeclampsia, smoking in pregnancy, neonatal morbidity, and perinatal corticosteroid therapy were not. In multivariate regression analyses, systolic BP decreased by 0.10 SD (P=0.08) and diastolic BP by 0.09 SD (P=0.02) for each week-longer gestation. Conclusions-Six-year-old children born extremely preterm have normal but slightly higher BP than their peers born at term. Although this finding is reassuring for children born preterm and their families, follow-up at older age is warranted.
  • Virtanen, Sonja; Kapp, Karmen; Rautamo, Maria M; Lotta, Schepel; Linden-Lahti, Carita; Cruz, Cristina D.; Tammela, Päivi (2021)
    Parenteral products must be compounded using an aseptic technique to ensure sterility of the medicine. We compared the effect of three clinical environments as compounding areas as well as different aseptic techniques on the sterility of the compounded parenteral product. Clinical pharmacists and pediatric nurses compounded 220 samples in total in three clinical environments: a patient room, a medicine room and biological safety cabinet. The study combined four methods: observation, environmental monitoring (settle plates), monitoring of personnel (finger dab plates) and sterility testing (membrane filtration). Of the compounded samples, 99% were sterile and no significant differences emerged between the clinical environments. Based on the settle plates, the biological safety cabinet was the only area that fulfilled the requirements for eliminating microbial contamination. Most of the steps on the observation form for aseptic techniques were followed. All participants disinfected their hands, wore gloves and disinfected the septum of the vial. Non-contaminated finger dab plates were mostly detected after compounding in the biological safety cabinet. Aseptic techniques were followed relatively well in all environments. However, these results emphasize the importance of good aseptic techniques and support the recommendation of compounding parenteral products in biological safety cabinets in clinical environments.
  • Parnanen, Katariina M. M.; Hultman, Jenni; Markkanen, Melina; Satokari, Reetta; Rautava, Samuli; Lamendella, Regina; Wright, Justin; McLimans, Christopher J.; Kelleher, Shannon L.; Virta, Marko P. (2022)
    Background Infants are at a high risk of acquiring fatal infections, and their treatment relies on functioning antibiotics. Antibiotic resistance genes (ARGs) are present in high numbers in antibiotic-naive infants' gut microbiomes, and infant mortality caused by resistant infections is high. The role of antibiotics in shaping the infant resistome has been studied, but there is limited knowledge on other factors that affect the antibiotic resistance burden of the infant gut. Objectives Our objectives were to determine the impact of early exposure to formula on the ARG load in neonates and infants born either preterm or full term. Our hypotheses were that diet causes a selective pressure that influences the microbial community of the infant gut, and formula exposure would increase the abundance of taxa that carry ARGs. Methods Cross-sectionally sampled gut metagenomes of 46 neonates were used to build a generalized linear model to determine the impact of diet on ARG loads in neonates. The model was cross-validated using neonate metagenomes gathered from public databases using our custom statistical pipeline for cross-validation. Results Formula-fed neonates had higher relative abundances of opportunistic pathogens such as Staphylococcus aureus, Staphylococcus epidermidis, Klebsiella pneumoniae, Klebsiella oxytoca, and Clostridioides difficile. The relative abundance of ARGs carried by gut bacteria was 69% higher in the formula-receiving group (fold change, 1.69; 95% CI: 1.12-2.55; P = 0.013; n = 180) compared to exclusively human milk-fed infants. The formula-fed infants also had significantly less typical infant bacteria, such as Bifidobacteria, that have potential health benefits. Conclusions The novel finding that formula exposure is correlated with a higher neonatal ARG burden lays the foundation that clinicians should consider feeding mode in addition to antibiotic use during the first months of life to minimize the proliferation of antibiotic-resistant gut bacteria in infants.
  • Junkkari, Ella; Hukkinen, Maria; Merras-Salmio, Laura; Koivusalo, Antti; Pakarinen, Mikko (Helsingin yliopisto, 2020)
    Objective. Recent studies have reported an increasing incidence of acute pancreatitis (AP) in children. The etiology of AP in children is more diverse compared to adults. All patients treated for acute pancreatitis (AP) or acute recurrent pancreatitis (ARP) in Helsinki University Children’s hospital during 1999-2018 were reviewed. Methods. ARP was considered as two or more episodes of AP over a lifetime. Demographics, clinical findings, laboratory test results, genetic assessment, imaging findings, endoscopic and surgical treatment, duration of hospital stay, number of pancreatitis episodes, and outcome were analyzed. Results. Of a total of 34 identified patients [n=22 (64%) AP; n=12 (35%) ARP; n=17 (50%) females] none died. The most frequent etiologies were pancreaticobiliary (26%), drug-induced (21%), hereditary (18%), autoimmune (9%) and idiopathic (21%) pancreatitis. An underlying SPINK1 (n=4) and PRSS1 mutation was found in five (15%) patients. Median age at diagnosis was 9.8 (8.2-11) years. Patients with pancreaticobiliary pancreatitis were younger at presentation [4.3 (2.5-9.8) vs. 10 (8.5-12) years, p=0.025] and underwent ERCP (n=7/9 vs. 8/25, p=0.025), and surgical or endoscopic interventions (n= 8/9 vs. 8/25 p=0.006) more frequently compared to the rest of the cohort. The most common long-term complications affecting 29% of patients were chronic upper abdominal pain and diarrhea, occurring each in three patients (8.8%), respectively. Four patients received pancreatic enzyme substitution, while none developed diabetes. Conclusions. Our study highlights the diverse etiology of pediatric pancreatitis necessitating comprehensive diagnostic work-up and management options with relatively low long-term morbidity.
  • Voutilainen, Silja H.; Kosola, Silja K.; Lohi, Jouko; Jahnukainen, Timo; Pakarinen, Mikko P.; Jalanko, Hannu J (2021)
    Background Unexplained graft fibrosis and inflammation are common after pediatric liver transplantation (LT). Objective We investigated the graft expression of fibrogenic genes and correlated the findings with transplant histopathology and outcome. Methods Liver biopsies from 29 recipients were obtained at a median of 13.1 (IQR: 5.0-18.4) years after pediatric LT. Control samples were from six liver-healthy subjects. Hepatic expression of 40 fibrosis-related genes was correlated to histological findings: normal histology, fibrosis with no inflammation, and fibrosis with inflammation. Liver function was evaluated after a subsequent follow-up of 9.0 years (IQR: 8.0-9.4). Results Patients with fibrosis and no inflammation had significantly increased gene expression of profibrotic TGF-beta 3 (1.17 vs. 1.02 p = .005), CTGF (1.64 vs. 0.66 p = .014), PDGF-alpha (1.79 vs. 0.98 p = .049), PDGF -beta (0.99 vs. 0.76 p = .006), integrin-subunit-beta 1 (1.19 vs. 1.02 p = .045), alpha-SMA (1.12 vs. 0.58 p = .013), type I collagen (0.82 vs. 0.53 p = .005) and antifibrotic decorin (1.15 vs. 0.99 p = .045) compared to patients with normal histology. mRNA expression of VEGF A (0.84 vs. 1.06 p = .049) was lower. Only a few of the studied genes were upregulated in patients with both fibrosis and inflammation. The gene expression levels showed no association with later graft outcome. Conclusions Altered hepatic expression of fibrosis-related genes is associated with graft fibrosis without concurrent inflammation.
  • Owall, Louise; Darvann, Tron A.; Hove, Hanne B.; Heliövaara, Arja; Duno, Morten; Kreiborg, Sven; Hermann, Nuno (2021)
    Objective: To quantify soft tissue facial asymmetry (FA) in children with nonsyndromic and Muenke syndrome-associated unicoronal synostosis (NS-UCS and MS-UCS), hypothesizing that MS-UCS presents with significantly larger FA than NS-UCS. Design: Retrospective cohort study. Patients and Methods: Twenty-one children (mean age: 0.6 years; range: 0.1-1.4 years) were included in the study (NS-UCS = 14; MS-UCS = 7). From presurgical computed tomography scans, facial surfaces were constructed for analysis. A landmark guided atlas was deformed to match each patient's surface, obtaining spatially detailed left-right point correspondence. Facial asymmetry was calculated in each surface point across the face, as the length (mm) of an asymmetry vector, with its Cartesian components providing 3 directions. Mean FA was calculated for the full face, and the forehead, eye, nose, cheek, mouth, and chin regions. Results: For the full face, a significant difference of 2.4 mm (P = .001) was calculated between the 2 groups, predominately in the transverse direction (1.5 mm; P < .001). The forehead and chin regions presented with the largest significant difference, 3.5 mm (P = .002) and 3.2 mm (P < .001), respectively; followed by the eye (2.4 mm; P = .004), cheek (2.2 mm; P = .004), nose (1.7 mm; P = .001), and mouth (1.4 mm; P = .009) regions. The transverse direction presented with the largest significant difference in the forehead, chin, mouth, and nose regions, the sagittal direction in the cheek region, and the vertical direction in the eye region. Conclusions: Muenke syndrome-associated unicoronal synostosis presented with significantly larger FA in all regions compared to NS-UCS. The largest significant differences were found in the forehead and chin regions, predominantly in the transverse direction.
  • Hämäläinen, Anssi-Petteri (Helsingfors universitet, 2010)
    IBD- ja reumasairaudet ovat elämänlaatua merkittävästi heikentäviä, kroonisia tulehduksellisia sairauksia, joiden keskivaikeiden ja vakavien muotojen hoidossa suoneen annettava biologinen TNF-alfa -tulehdustekijän vasta-aine infliksimabi (Remicade®) on vankassa asemassa. Infliksimabin hyvän tehon kääntöpuolena esiintyy yleisesti haittoja, joista infuusion aikana tai pian sen jälkeen ilmenevät allergistyyppiset reaktiot ovat hoitoa hankaloittava ja jopa vaarallinen alaluokka. Infuusioreaktioiden estoon ei nykyisellään ole todistettavasti tehokkaita keinoja. Parasetamoli ja setiritsiini osoittautuivat tässä käyttötarkoituksessa tehottomiksi. HUS:n Lasten ja nuorten sairaalassa aloitettiin 11.3.2009 hoitokokeilu asetosalisyylihapolla (ASA, Disperin®), annosteltuna painonmukaisesti per os, päämääränä selvittää prospektiivisesti ASA:n käyttömahdollisuudet infuusioreaktioiden ehkäisyssä. Tämän tutkielman aineisto kerättiin esilääkekokeilun alun ja 24.6.2010 välisellä ajalla (yhteensä 67 viikkoa) infliksimabi-infuusiossa Lasten ja nuorten sairaalan osasto 2:lla käyneiden IBD- ja reumapotilaiden asiakirjoista. Vain 1 (0,2 %) ASA:n kanssa annetuista infuusioista johti infuusioreaktioon kun aiemmin parasetamolin ja setiritsiinin kanssa todettiin 11 (2,9 %) reaktiota. GraphPad Prism 5 -ohjelmistolla tehdyn tilastoanalyysin perusteella tulokset osoittavat ASA:n olevan erittäin lupaava infuusioreaktioiden estolääke.
  • Vironen, Aleksi (Helsingin yliopisto, 2021)
    Evidence based medicines alongside with age-appropriate dosage forms are essential in enabling appropriate treatment for any patient group. Pediatric pharmacotherapy, however, is lacking age-appropriate dosage forms and research-based evidence regarding the dosing, efficacy, and safety of medicines. Orally administered drugs require manipulation to enable administration and are often used against the indications approved in the marketing authorization and summary of product characteristics (SmPC). This off-label use puts pediatric patients at risk for medicational errors and adverse drug reactions. The aim of this study was to investigate recent trends in oral dosage forms used in pediatric randomized controlled trials (RCTs), with emphasis on age appropriateness. The results could be utilized in developing evidence-based dosage forms, suitable for all pediatric patients aged 0-17 years, and manufacturable in a small scale in a hospital pharmacy. This study was conducted as a systematic review following the PRISMA Statement. The literature search was carried out from Pubmed and Scopus databases and it covered a five-year period of 2015-2020. References from the databases were entered to the Covidence systematic review platform. After removing duplicates 3333 articles were left for screening. Two independent researchers selected the articles first screening by title and abstract, and then by full text review. A qualitative content analysis was conducted on the included articles. Altogether 77 articles met the inclusion criteria. Dosage forms included were tablets (n=37), liquids (n=21), capsules (n=18) and multiparticulates (n=6). Majority of the dosage forms were conventional (n=49) compared to more advanced novel modified release and fixed-dose combination formulations (n=33). Based on our results, orally administered dosage forms used in the recent pediatric RCTs are still limited by poor acceptability, palatability, and the need to manipulate dosage forms to enable administration. These issues are similar to the ones related to the off-label use of medicine that compromise patient safety. Majority of the dosage forms included in our study were tablets, indicating a positive shift towards more safe and acceptable dosage form. Formulations were also evolving towards dispersible, extended-release and fixed-dose combinations that offer additional benefits for pediatric patients. The low number of children < 2 years old included in study populations and the poor acceptability profile reported by most studies limit our conclusions on an ideal age-appropriate dosage form. Further research is needed on unifying the guidelines used in pediatric drug development.
  • Malmberg, Maiju; Malmberg, L. Pekka; Pelkonen, Anna S.; Makela, Mika J.; Kotaniemi-Syrjänen, Anne (2021)
    Background The objective of this study was to evaluate the role of body mass index with regard to exercise performance, exercise-induced bronchoconstriction (EIB), and respiratory symptoms in 7- to 16-year-old children. Methods A total of 1120 outdoor running exercise challenge test results of 7- to 16-year-old children were retrospectively reviewed. Lung function was evaluated with spirometry, and exercise performance was assessed by calculating distance per 6 minutes from the running time and distance. Respiratory symptoms in the exercise challenge test were recorded, and body mass index modified for children (ISO-BMI) was calculated for each child from height, weight, age, and gender according to the national growth references. Results Greater ISO-BMI and overweight were associated with poorer exercise performance (P <.001). In addition, greater ISO-BMI was independently associated with cough (P = .002) and shortness of breath (P = .012) in the exercise challenge. However, there was no association between ISO-BMI and EIB or with wheeze during the exercise challenge. Conclusion Greater ISO-BMI may have a role in poorer exercise performance and appearance of respiratory symptoms during exercise, but not in EIB in 7- to 16-year-old children.
  • Malmberg, Maiju; Malmberg, Pekka; Pelkonen, Anna; Mäkelä, Mika; Kotaniemi-Syrjänen, Anne (Helsingin yliopisto, 2020)
    Tämän tutkimuksen tarkoitus oli arvioida painoindeksin yhteyttä fyysiseen suorituskykyyn, rasitusastmareaktioon ja rasituksen laukaisemiin hengitystieoireisiin (yskään, vinkunaan ja hengenahdistukseen) kouluikäisillä lapsilla. 1120 ulkojuoksukokeen tulokset käytiin retrospektiivisesti läpi. Nämä ulkojuoksukokeet oli suoritettu Iho- ja allergiasairaalassa osana kouluikäisten lasten tavanomaisia astmaselvittelyjä. Keuhkojen toimintaa arvioitiin spirometrialla. Astmalle diagnostisen rasituksen laukaiseman keuhkoputkien supistumisen eli rasitusastmareaktion rajana pidettiin vähintään 15 prosentin laskua uloshengityksen sekuntikapasiteetissa rasituksen jälkeen. Fyysinen suorituskyky arvioitiin laskemalla todellisesta juoksuajasta ja -matkasta 6 minuutissa juostu matka. Hengitystieoireet ja -löydökset kirjattiin kokeen aikana, ja lasten ikään ja sukupuoleen suhteutettu painoindeksi eli ISO-BMI laskettiin kansallisia kasvuviitearvoja käyttäen. Suurempi ISO-BMI ja ylipaino ennustivat heikompaa fyysistä suorituskykyä. Lisäksi suurempi ISO-BMI oli yhteydessä yskään ja hengenahdistukseen rasituskokeen aikana. Sen sijaan ISO-BMI ei liittynyt rasitusastmareaktioon eikä vinkunaan. Tämän tutkimuksen löydösten perusteella kouluikäisillä lapsilla ylipaino voi ennustaa heikompaa fyysistä suorituskykyä ja rasitusperäisten hengitystieoireiden ilmaantumista, mutta ei rasitusastmareaktiota. Mikäli asianmukaisia keuhkojen toimintatutkimuksia ei suoriteta, voidaan astmadiagnoosi asettaa väärin perustein. Tutkimus on toistaiseksi suurin kouluikäisten lasten ylipainon, rasitusastmareaktion ja fyysisen suorituskyvyn yhteyttä tarkasteleva tutkimus, mutta koska tutkimus oli luonteeltaan retrospektiivinen asiakirjatutkimus, tulokset tulisi jatkossa vahvistaa prospektiivisella seurantatutkimuksella.
  • European Soc Blood Marrow Transpla; Lawitschka, Anita; Lucchini, Giovanna; Vettenranta, Kim; Corbacioglu, Selim (2020)
    Pediatric allogeneic hematopoietic cell transplantation (HCT) practices differ from those of adults, particularly the heterogeneity of transplantable nonmalignant diseases and the lower incidence of graft-versus-host disease (GVHD). Several guidelines regarding the management of acute (a) GVHD in adult HCT have been published. We aimed to capture the real-life approaches for pediatric aGVHD prophylaxis/treatment, and data from 75/193 (response rate 39%) EBMT centers (26 countries) were included, representing half (48%) of the pediatric EBMT-HCT activity. Results with >= 75% approval from respondents (74/75) for GVHD prophylaxis after myeloablative HCT for malignancies partially contradict published guidelines: Single-agent cyclosporine A (CsA) was used for matched sibling donor HCT in 47%; blood CsA levels were reported lower; the relapse risk in malignant diseases influenced GVHD prophylaxis with early withdrawal of CsA; distinct longer duration of CsA was employed in nonmalignant diseases. Most centers used additional anti-thymocyte globulin for matched unrelated and mismatched donor HCT, but not for matched siblings. Regarding prophylaxis in nonmyeloablative conditioning (mainly for nonmalignant diseases), responses showed broad heterogeneity. High conformity was found for first-line treatment; however, results regarding steroid-refractory aGVHD indicate an earlier diagnosis in children. Our findings highlight the need for standardized pediatric approaches toward aGVHD prophylaxis/treatment differentiated for malignant and nonmalignant underlying diseases.
  • Kaskinen, Anu; Ayeboa-Sallah, Benjamin; Teivaanmäki, Tiina; Wärnhjelm, Elina; Korhonen, Liisa; Helve, Otto (2018)
    Background: Pediatric physician-led Web-based chat services offer a novel, low-threshold communication channel between caregivers and physicians. Objective: Our aim was to describe chat conversations between caregivers and physicians in a Web-based chat service to determine the factors that should be considered when planning a similar chat service. We also aimed to evaluate whether caregivers considered the consultations helpful, whether physicians considered they could answer caregivers' questions, and whether further face-to-face medical contact was needed. Methods: In September 2015, a private medical center for children in the greater Helsinki area initiated a Web-based chat service, accessible via any device with an internet connection, open from 9 am to 9 pm local time. Four residents in pediatrics, who had performed at least 60% of their 6-year residency program, served as the physicians responsible for chat consultations with caregivers of children. Between October 2015 and March 2016, 343 consecutive consultations were immediately evaluated by a chat physician. On average, caregivers were followed up by email questionnaire 7-14 days later, which 98 caregivers answered a median of 11 (interquartile range, IQR, 7-20) days later. Results: The age of the children whose caregivers contacted the chat service was a median of 2.1 (IQR 0.83-4.69) years, and 29.8% (102/342) of the children were less than 1 year old. The majority (119/343, 34.7%) of the chat conversations took place from 9 am to noon, and infections were the most common concern in over half of cases (189/343, 55.1%). Chat physicians recommended a face-to-face appointment with a physician for that same day in 13.7% (47/343) of the cases. A face-to-face exam was recommended for that same day more often if the chat concerned infection (36/189, 19.0% cases) compared with other reasons (11/154, 7.1%, cases; P=. 001). Physicians felt capable of answering caregivers' questions in 72.6% (249/343) of the cases, whereas 93% (91/98) of caregivers considered physicians' answers helpful. Whether caregivers had to take their children to see a physician that same day or whether caregivers' main concern was infection was not found to be associated with whether caregivers considered physicians' answers helpful or not. However, physicians felt more capable of answering caregivers' questions when the main concern was infection. Conclusions: Parental consultations via Web-based chat service often take place before noon and focus on infection-related issues as well as on the health and illness of very young children. These factors should be considered when planning or setting up such a service. Based on the high satisfaction with the chat service by both physicians and caregivers, Web-based chat services may be a useful way to help caregivers with concerns about their child's health or illness.
  • Aaltonen, Venla (Helsingin yliopisto, 2020)
    PExA on uudenlainen ja kajoamaton pienten hengitysteiden tutkimusmenetelmä. Tutkimuk-semme tarkoituksena oli selvittää menetelmän soveltuvuutta astman diagnostiikassa lapsilla ja nuorilla. PExA-tutkimuksia ei ole aiemmin tehty lapsipotilailla. Satunnaisesti valitut tutkimushenkilöt olivat 4-18-vuotiaita, puolet tyttöjä ja puolet poikia. Ast-maatikkojen ryhmään kuului lapsia, joilla oli joko lääkärin diagnosoima astma tai oireita, joiden vuoksi he olivat astmaselvittelyissä. Kolmasosa tutkimushenkilöistä oli terveitä verrokkeja. Tutkimuksessamme arvioimme PExA-mittauksen kliinisen soveltuvuuden ja toistettavuuden lisäksi mittaustulosten eroja astmaatikoiden ja terveiden verrokkien välillä. Tarkasteltavia muuttujia olivat uloshengitysilman partikkeleiden massa, uloshengitetyn ilman kokonaistila-vuus ja ilmavirtauksen nopeus. PExA-mittaus tehtiin sekä ennen että jälkeen bronkodilaatio-testin. Suurin osa (88%) tutkimushenkilöistä suoritti PExA-mittauksen onnistuneesti. Nuorin tekni-sesti hyväksytyn mittauksen suorittanut oli neljävuotias, mutta vain harva alle kuusivuotias onnistui hengitystekniikan toteutuksessa. Havaitsimme, että uloshengitysilman partikkelien massa sekä massa uloshengitetyn ilman kokonaistilavuuden suhteen olivat terveillä verro-keilla suurempia kuin astmaatikoilla. PExA-menetelmä soveltui onnistuneesti terveiden lasten sekä astmaatikoiden kliiniseen tut-kimukseen 6-18-vuotialla. PExA 2.0 vaikuttaisi erottelevan terveet lapset astmaatikoista jo ensimmäisten puhallusten perusteella.
  • Tuokkola, J; Heikkila, A; Junttila, K; Orell, H (2021)
    Background Malnutrition is related to an increased rate of complications and prolonged hospital stays. Malnutrition risk screening is recommended for all hospital inpatients, but its applicability as part of routine care is not well known. Methods The prevalence of malnutrition risk, measured by the Screening Tool for Risk of Impaired Nutritional Status and Growth (STRONGkids), and acute malnutrition were studied 1 day per year in all pediatric inpatient and day wards in 2015 and 2016 and in inpatients and outpatients in 2017 at a university hospital. Nurses carried out the STRONGkids screening and measured the weight and height of each child, recording the information in the hospital database, where it was subsequently extracted along with data on the child's diagnoses, procedures, and the length and costs of hospital stay. Results In all, 696/1217 patients (57.2%) were screened. Of inpatients and outpatients, 37/398 (9.3%) and 3/298 (1.0%), respectively, were classified as being at high risk of malnutrition. The corresponding figures for those who were acutely malnourished were 18/260 (6.9%) and 11/264 (4.2%). High risk of malnutrition increased the costs and length of hospital stay (P < .05). Dietetic input was involved during hospital stay in 12/37 (32.4%), 32/173 (18.5%), and 13/188 (6.9%) of inpatients at high, moderate, and low risk, respectively (P < .001). Nutrition risk screening did not detect all patients classified as acutely malnourished. Conclusion Hospitals need to ensure proper application of nutrition screening, develop a protocol for the care of children at nutrition risk, and monitor the use of this protocol.
  • Saari, Viivi (Helsingin yliopisto, 2021)
    Autoimmuunipolyendokrinopatia-kandidiaasi-ektodermidystrofia (APECED) on harvinainen perinnöllinen suomalaiseen tautiperintöön kuuluva autoimmuunisairaus. Taudin tyypillisiä ilmentymiä ovat pitkäaikaiset kandidainfektiot, lisämunuaiskuoren vajaatoiminta ja lisäkilpirauhasten vajaatoiminta. Kolmanneksi yleisin hormonivaje naisilla on ennenaikainen munasarjojen vajaatoiminta, jonka seurauksia ovat kuukautisten puuttuminen, vaihdevuosioireet sekä estrogeenipuutoksen aiheuttamat pitkäaikaiset terveyshaitat. Tutkimuksen tavoitteena oli kuvata APECED-tautia sairastavien naispotilaiden murrosiän kehitys, kasvu murrosiän aikana ja ennenaikaisen munasarjojen vajaatoiminnan ilmaantuminen. Tutkimukseen kerättiin tietoja yhteensä 40 naispotilaasta suomalaisesta APECED-kohortista. Tutkimukseen otettiin mukaan yli 12-vuotiaat potilaat, joiden munasarjojen toiminnasta oli kerättävissä riittävästi tietoa sairaskertomuksista vuosilta 1968-2018. Ennenaikaisen munasarjojen vajaatoiminnan diagnoosi perustui vaihdevuosioireisiin, kuukautisten poisjäämiseen ja/tai FSH-arvoon ≥ 40 IU/L ennen 40 v ikää. Murrosikä alkoi spontaanisti 34 potilaalla, joista 29:llä kuukautiset alkoivat spontaanisti. Yhdellätoista potilaalla tarvittiin hormonikorvaushoitoa murrosiän kehitystä varten. Munasarjojen vajaatoiminta kehittyi yhteensä 28 potilaalle (70%) 16.0 vuoden mediaani-iässä. Munasarjojen vajaatoimintaa sairastavat potilaat olivat lyhyempiä menarken aikaan kuin muut APECED-potilaat, mutta aikuispituus ei eronnut ryhmien välillä. Munasarjojen vajaatoimintaa sairastavilla oli merkittävästi useammin lisämunuaiskuoren vajaatoiminta (93% vs. 58%, p = 0.017) ja munasarjavasta-aineita (81% vs. 30%, p = 0.003) kuin muilla APECED-potilailla (n = 12). Harvat potilaista olivat saaneet lapsia, mutta hedelmällisyydessä ei havaittu eroa munasarjojen vajaatoimintaa sairastavien ja muiden naispotilaiden välillä. Ennenaikainen munasarjojen vajaatoimina kehittyi suurimmalle osalle APECED-potilaista ja useimmiten pian menarken jälkeen. Hormonikorvaushoidon oikea-aikainen aloittaminen on tärkeää normaalin murrosiän kehityksen ja kasvun turvaamiseksi. Mahdollisuuksia hedelmällisyyden säilyttämiseen ennen munasarjojen vajaatoiminnan kehittymistä tulee pyrkiä kehittämään.
  • Kocoshis, Samuel A.; Merritt, Russell J.; Hill, Susan; Protheroe, Susan; Carter, Beth A.; Horslen, Simon; Hu, Simin; Kaufman, Stuart S.; Mercer, David F.; Pakarinen, Mikko P.; Venick, Robert S.; Wales, Paul W.; Grimm, Andrew A. (2020)
    Background This study evaluated the safety and efficacy of teduglutide in pediatric patients with short bowel syndrome-associated intestinal failure (SBS-IF). Methods A 24-week, phase III trial with 2 randomized, double-blind teduglutide dose groups and a nonblinded standard of care (SOC) arm was used; patients received 0.025 mg/kg or 0.05 mg/kg teduglutide once daily. Safety end points included treatment-emergent adverse events (TEAEs) and growth parameters. The primary efficacy/pharmacodynamic end point was the number of patients who achieved a >= 20% reduction in parenteral support (PS) from baseline at week 24. Results All 59 enrolled patients completed the study (0.025 mg/kg, n = 24; 0.05 mg/kg, n = 26; SOC, n = 9). Baseline demographics and disease characteristics were comparable among groups. TEAEs were reported by 98% and 100% of patients in the teduglutide and SOC groups, respectively. The most common TEAEs in the teduglutide-treated groups were pyrexia and vomiting. The primary end point was achieved by 13 (54.2%), 18 (69.2%), and 1 (11.1%) patients who received 0.025 mg/kg teduglutide, 0.05 mg/kg teduglutide, and SOC, respectively (P <0.05 vs SOC). Both 0.025-mg/kg and 0.05-mg/kg teduglutide groups showed clinically significant reductions in PS volume (P <0.05 vs SOC), PS calories, days per week and hours per day of PS infusions, and increases in enteral nutrition and plasma citrulline at week 24 compared with baseline. Two (8.3%, 0.025 mg/kg teduglutide) and 3 patients (11.5%, 0.05 mg/kg teduglutide) achieved enteral autonomy. Conclusion The safety profile of teduglutide was similar to that reported previously in children and adults. Treatment with teduglutide was associated with significant reductions in PS for pediatric patients with SBS-IF over 24 weeks.