bluuebunny/crossref_metadata_2025_split huggingface dataset:
ChemInform Abstract: DIRECT NITROSATION OF ANILINE DERIVATIVES AND OTHER NUCLEOPHILIC SPECIES BY N‐NITROSODIPHENYLAMINE
Authors: THOMPSON J. T. , WILLIAMS D. L. H. 
Published: April 2016

AbstractIn wäßriger schwefelsaurer Lösung wird die Titelverbindung (I) in Gegenwart von überschüssigem Na‐azid zum Amin (II) denitrosiert.
ChemInform Abstract: 3,3‐DIMETHYLALLENYLLITHIUM. REACTION WITH ELECTROPHILES LEADING TO CARBENOID, ELECTRON TRANSFER, AND NUCLEOPHILIC PROCESSES
Authors: CREARY X. 
Published: April 2016

AbstractBei Reaktion des Allens (I) mit den Benzylchloriden (II) in Gegenwart von LiTMP können als Hauptprodukte die Methylencyclopropane (III) und (IV) sowie die Acetylene (V) isoliert werden.
ChemInform Abstract: REACTION OF ETHYL ACETOACETATE WITH ISOQUINOLINIUM AND PYRIDINIUM YLIDES
Authors: KATO T. , CHIBA T. , TANAKA S. , SASAKI T. 
Published: April 2016

AbstractAcetessigsäureethylester liefert mit den Yliden (I), (VII) und (X) die Cyclisierungsprodukte (III)‐(VI), (VIII) und (IX) bzw. (XI).
ChemInform Abstract: REACTIONS OF BISHALOMETHYLARENES WITH SUPEROXIDE
Authors: DAO L. H. , HOPKINSON A. C. , LEE‐RUFF E. , RIGAUDY J. 
Published: April 2016

AbstractAus den Bis‐halogenmethylaromaten (I), (V) und (VIII) entstehen bei Reaktion mit K‐superoxid die Dioxene (II), (VI) und (IX).
ChemInform Abstract: STUDIES ON THE SYNTHESES OF HETEROCYCLIC COMPOUNDS. DCCXXVIII. A SIMPLE SYNTHESIS OF 1,3‐BENZOAXAZIN‐4‐ONES FROM SALICYLIC ACID
Authors: KAMETANI T. , HIGA T. , LOC C. V. , IHARA M. , FUKUMOTO K. 
Published: April 2016

AbstractSalicylsäurechlorid ergibt mit dem Dihydrocarbolin (II) das Cyclisierungsprodukt (III).
ChemInform Abstract: SYNTHESIS AND STRUCTURE OF AROMATIC AND HETEROCYCLIC TELLURIUM COMPOUNDS. XIII. SYNTHESIS AND STRUCTURE OF NEW STABLE TELLURIUM YLIDES
Authors: SADEKOV I. D. , USACHEV A. I. , BREN' V. A. , KOROBOV M. S. , TSEIMAKH I. D. , MINKIN K. I. 
Published: April 2016

AbstractDiorgano‐tellurdihalogenide (I) reagieren mit Dimedon (II) in Gegenwart von Triethylamin zu den stabilen Yliden (III).
ChemInform Abstract: MERCURY(II) COMPLEXES WITH SOME DITHIOUREAS
Authors: MOHAPATRA B. B. , PAUL B. , MOHAPATRA B. K. , GURU S. 
Published: April 2016

AbstractDurch Reaktion von Arylisothiocyanaten bzw. Ammoniumthiocyanaten mit entsprechenden Diaminverbindungen werden die Bis‐[thioharnstoff]‐Derivate dargestellt.
ChemInform Abstract: NICKEL(II) COMPLEXES OF THIOHYDRAZONATES. I
Authors: GABEL J. , LARSEN E. , TRINDERUP P. 
Published: April 2016

AbstractZur Darstellung neuer Kondensationsprodukte von Thiohydraziden und Mono‐ bzw. Diketonen als Liganden in Nickel(II)‐Innerkomplexen können zwei Methoden angewandt werden: 1. Die Reaktion der Thioamide (I) mit Hydrazinium‐ und N i(II)‐Ionen bei pH N 4 in Gegenwart der Ketone (II) und 2. die Kondensation der Ni‐Komplexe (IV) mit den Ketonen (II) in Gegenwart katalytischer Mengen HCl.
ChemInform Abstract: COPPER(II) AND NICKEL(II) COMPLEXES WITH SCHIFF BASES DERIVED FROM 2‐HYDROXYPROPIOPHENONE
Authors: JANI B. , BHATTACHARYA P. K. 
Published: April 2016

AbstractNach bekannter Methode werden die binären Komplexe (III) und die Gemischt‐ Ligand‐Komplexe (VI) der Schiffschen Basen dargestellt.
ChemInform Abstract: SOME COMPLEXES OF PLATINUM, PALLADIUM, RHODIUM, AND IRIDIUM FORMED FROM THE BULKY LIGAND (PHENYLETHYNYL)DI‐TERT‐BUTYLPHOSPHINE
Authors: EMPSALL H. D. , HYDE E. M. , MENTZER E. , SHAW B. L. 
Published: April 2016

AbstractDer aus Di‐tert.‐butylchlorphosphin und 2‐Phenylethinyl‐Li synthetisierte Titelligand (Ausb. 80%) reagiert bei 2:1‐molarer Umsetzung mit den Nitril‐Komplexen (I) zu den als Konformerengemisch isolierten trans‐Diphosphin‐Komplexen (IIa).
ChemInform Abstract: IRIDIUM AND RHODIUM NITROSYL O‐CATECHOLATO METAL COMPLEXES
Authors: GHEDINI M. , DENTI G. , DOLCETTI G. 
Published: April 2016

AbstractAlkoholische Suspensionen der dikationischen Nitrosylkomplexe (I) reagieren mit stöchiometrischen Mengen des entsprechenden Brenzcatechins in Gegenwart stöchiometrischer Mengen NaOH in Abhängigkeit vom Metall und von den Substituenten zu den neutralen pentaund tetrakoordinierten Komplexen (II) und (III).
ChemInform Abstract: CHEMISTRY OF BIS(CYCLOOCTA‐1,5‐DIENE)PLATINUM‐ REACTIONS WITH ELECTROPHILES
Authors: GREEN M. , GROVE D. M. , SPENCER J. L. , STONE F. G. A. 
Published: April 2016

AbstractBei Reaktion des Titelkomplexes mit Acetylaceton wird der Komplex (I), mit Hexa‐ fluoracetylaceton der Komplex (II) als Hauptprodukt neben (III) und mit Trifluoracetylaceton ein Isomerengemisch (IV) mit einer Gesamtausb.
ChemInform Abstract: STEREOSPECIFIC TOTAL SYNTHESIS OF DL‐HASTANECINE AND DL‐DIHYDROXYHELIOTRIDANE
Authors: DANISHEFSKY S. , MCKEE R. , SINGH R. K. 
Published: April 2016

AbstractDie Darstellung von dl‐Hastanecin (Ia) erfolgt ausgehend vom Aldehyd (IIb), erhältlich durch Ozonolyse von (IIa); das Reduktionsprodukt (IVb) von (IVa) wird dann suk‐ ′ zessiv in das Acylierungsprodukt (IVc), die Diazoverbindung (IVd) (bekannte Reaktion), das Cyclopropan (V) und das Diacetoxylactam (VI) in (Ia) übergeführt.
ChemInform Abstract: A CONVENIENT SYNTHESIS OF 1‐(2′,3′‐DIDEOXY‐β‐D‐ERYTHRO‐HEX‐2′‐ENOPYRANOSYL)URACIL
Authors: YAMAZAKI T. , SUGIYAMA H. , MATSUDA K. , SETO S. 
Published: April 2016

AbstractDas Glucopyranosyluracil (I) reagiert mit Benzaldehyd zu dem Benzyliden‐Derivat (IIa), dessen Mesylat (IIb) in das Anhydronucleosid (III) übergefihrt wird.
ChemInform Abstract: SYNTHESIS OF N6‐ OR 8‐SUBSTITUTED 9‐(β‐D‐ARABINOFURANOSYL)ADENINES AND THEIR ANTIVIRAL ACTIVITIES AGAINST HERPES SIMPLEX AND VACCINIA VIRUSES
Authors: KANEKO M. , KIMURA M. , NISHIMURA T. , SHIMIZU B. 
Published: April 2016

AbstractÜber das 8,2′‐O‐Cycloadenosin (IV) erhält man durch Reaktion mit Schwefelwasserstoff und anschließende Entschwefelung mit Raney‐Nickel das Arabinofuransyladenosin (Vb) in 83%iger Ausbeute.
ChemInform Abstract: NATURALLY OCCURRING COMPOUNDS RELATED TO PHENALENONE. PART 8. STRUCTURE AND SYNTHESIS OF DEMETHYLHERQUEICHRYSIN
Authors: FROST D. A. , HALTON D. D. , MORRISON G. A. 
Published: April 2016

AbstractModifizierte Claisen‐Umlagerung des Allylethers (I) gibt das Dion (IIa), dessen Methylierung mit Diazomethan neben dem Enolether (III) das Tetramethoxyderivat (IIb) liefert.
ChemInform Abstract: SYNTHESIS OF PROSTAGLANDIN H2 METHYL ESTER
Authors: JOHNSON R. A. , NIDY E. G. , BACZYNSKYJ L. , GORMAN R. R. 
Published: April 2016

AbstractReaktion des in 5 Schritten aus dem PGFza‐ester (Ia) erhältlichen Ditosylats (Ib) gibt ein Gemisch der isomeren Dibromide (IIa), (IIb) und (IIc).
A phase I study of the investigational NEDD8-activating enzyme inhibitor pevonedistat (TAK-924/MLN4924) in patients with metastatic melanoma
Authors: Bhatia Shailender , Pavlick Anna C. , Boasberg Peter , Thompson John A. , Mulligan George , Pickard Michael D. , Faessel Hélène , Dezube Bruce J. , Hamid Omid 
Published: April 2016

SummaryPurpose The therapeutic index of proteasome inhibitors may be improved through selective inhibition of a sub-component of the ubiquitin-proteasome system, such as the NEDD8-conjugation pathway. This multicenter, phase I, dose-escalation study assessed safety and the maximum tolerated dose (MTD), pharmacokinetics, pharmacodynamics, and antitumor activity of pevonedistat, an investigational NEDD8-activating enzyme (NAE) inhibitor, in patients with metastatic melanoma. Methods Patients received intravenous pevonedistat on Days 1, 4, 8, 11 (schedule A) or 1, 8, 15 (schedule B) of 21-day cycles. Results 26 patients received pevonedistat 50–278 mg/m2 on schedule A; 11 patients received pevonedistat 157 mg/m2 on schedule B. The schedule A MTD was 209 mg/m2: dose-limiting toxicities (DLTs) included grade 3 hypophosphatemia and grade 3 increased blood creatinine (associated with grade 3 hyperbilirubinemia). Two schedule A patients experienced acute organ failure toxicities, one of whom experienced grade 5 acute renal failure. Dose escalation did not occur in schedule B: DLTs included grade 3 myocarditis, grade 2 acute renal failure, and grade 2 hyperbilirubinemia in a single patient. Pevonedistat pharmacokinetics were approximately dose-proportional across the dose range studied, with a biphasic disposition profile characterized by a short elimination half-life (~10 h). Pharmacodynamic studies showed increases in NAE-regulated transcripts post-treatment; all post-dose biopsy samples were positive for pevonedistat-NEDD8 adduct. One schedule A patient achieved a partial response; 15 patients had stable disease (4 lasting ≥6.5 months). Conclusions Pevonedistat was generally well tolerated at the MTD. Anticipated pharmacodynamic effects of NAE inhibition were observed with single-agent pevonedistat in peripheral blood and tumor tissue.
Genotype × environment interaction and selection for drought adaptation in sweetpotato (Ipomoea batatas [L.] Lam.) in Mozambique
Authors: Andrade Maria I. , Naico Abdul , Ricardo José , Eyzaguirre Raúl , Makunde Godwill S. , Ortiz Rodomiro , Grüneberg Wolfgang J. 
Published: April 2016

AbstractSweetpotato is grown throughout the year in Mozambique but drought affects storage root yield and biomass productivity. The objectives of this research were to estimate the impact of genotype × environment interactions (G × E) in sweetpotato and select genotypes based on drought indices such as geometric mean, percent yield reduction, drought sensitivity index and harvest index. A total of 58 clones were evaluated during the dry season of 2006, 2008 and 2009. Two treatments were applied for this multi-year trial: full irrigation and without irrigation at the middle of root initiation growth stage. The field layout was a randomized complete block design with three replications. ‘Jonathan’, ‘Resisto’ and ‘Tanzania’ were the check cultivars in each treatment. Storage root and vine yields were recorded at harvest in the trials. Harvest index was computed from the yield data. The analysis of variance, regression and the additive main effects multiplicative interaction (AMMI) analyses, plus phenotypic coefficient of variation and ecovalence were used for dissecting the G × E and assessing the stability of each clone. Treatment, genotype × treatment and genotype × year (G × Y) interactions had highest contributions to the variation in storage root yield observed among clones. The stability of harvest index was significantly correlated with the absolute AMMI’s IPCA1 and IPCA2 values for storage root yield. Cultivar performance varied within treatments. Four clones had significantly higher storage root yield (t ha−1) than ‘Tanzania’, the best check cultivar under drought. In conclusion, storage root yield (t ha−1) was negatively affected by drought and G × Y interaction. Harvest index stability and the geometric mean may be key to identify clones with storage root yield stability and high storage root yield under both treatments. At least two environments should be used at early breeding stages to consider harvest index in the early breeding cycle.
Lipids and Fatty Acid Profiling of Major Indian Garcinia Fruit: A Comparative Study and its Nutritional Impact
Authors: Patil Mahesh Mansing , Muhammed Ali M. , Anu‐Appaiah K. A. 
Published: April 2016

AbstractThe effects of plant oils on health are being intensively studied. Many fatty acids have attracted significant scientific attention since the studies pointed them as potential nutrients. An attempt was made to analyze the variation in three major Indian Garcinia fruits for their oils, lipid sub‐classes, fatty acids and total amino acids. Solvent extraction and chromatographic techniques were used for the isolation, purification, separation and detection of these compounds. Three major Garcinia fruits G. gummi‐gutta, G. indica and G. xanthochymus varied in their chemical composition. Oil content was significantly higher (p < 0.05) in G. xanthochymus seeds (16.9 %) when compared to 11.21 % in G. gummi‐gutta seeds. Fatty acids observed were mainly capric, undecanoic, lauric, palmitic, stearic, oleic and linolelaidic acid. Monounsaturated fatty acids (MUFA) were predominant in both pericarp and seeds with oleic acid being the major fraction (29.24‐58.6 %). The lipid classification of Garcinia oils showed the varying percentage of neutral lipids, glycolipids and phospholipids. Oleic acid (32.91–71.54 %) was found to be the major fatty acid in neutral‐, glyco‐ and phospho‐lipids. Alanine, leucine, proline and phenylalanine were the predominant amino acids found in Garcinia fruits. The study has broadened our understanding related to the different biochemical composition of Garcinia fruits, thereby providing the groundwork that may lead to the production, utilization and application of products from Garcinia in a more efficient way.
The value of three‐dimensional helical computed tomography for the retrograde flexible ureteronephroscopy in the treatment of lower pole calyx stones
Authors: Xu Yan , Lyu Jian‐Lin 
Published: April 2016

AbstractObjectiveThe aim of our study was to determine if there is any advantage of three‐dimensional helical computed tomography (3D‐HCT) over intravenous urogram (IVU) for the retrograde flexible ureteronephroscopy in the treatment of lower pole calyx stones.MethodsFrom June 2012 to January 2014, a total of 52 cases of lower pole renal stones underwent retrograde intrarenal surgery (RIRS) in our center. All patients underwent a preoperative IVU and three‐dimensional helical computed tomography urography (3D‐CTU) program to define the collecting system anatomy, manly concerning the following lower pole features; infundibu‐lopelvic angle (IPA), infundibular length (IL), and infundibular width (IW). The examinations were performed in the same center of reference with a standardized method and with 3D‐HCT Siemens Somaton Plus equipment. The measurements were performed by the same researcher, using a ruler and a square.ResultsBased on clinical threshold difference of the anatomic factors on an IVU image to compare the difference between an IVU image and a 3D‐CT image of 52 patients, the IPA was <30° when measured on intravenous pyelography (IVP) for 21 patients. We found that with the IPA of <30° measured with IVP only 19% (4/21) were correctly classified in the same size category using 3D‐HCT, whereas 81% (17/21) were upgraded to 40–50° on 3D‐CT. This difference was significant between IVP and 3D‐HCT.Conclusions3D‐HCT has advantages over IVU when analyzing the morphometric and the morphological features of kidney lower pole spatial anatomy for the retrograde flexible ureteronephroscopy in the treatment of lower pole calyx stones.
Abnormal frontostriatal connectivity in adolescent-onset schizophrenia and its relationship to cognitive functioning
Authors: James A. , Joyce E. , Lunn D. , Hough M. , Kenny L. , Ghataorhe P. , Fernandez H. , Matthews PM , Zarei M. 
Published: April 2016

AbstractBackgroundAdolescent-onset schizophrenia (AOS) is associated with cognitive impairment and poor clinical outcome. Cognitive dysfunction is hypothesised, in part, to reflect functional dysconnectivity between the frontal cortex and the striatum, although structural abnormalities consistent with this hypothesis have not yet been demonstrated in adolescence.ObjectiveTo characterise frontostriatal white matter (WM) tracts in relation to cognition in AOS.DesignA MRI volumetric and diffusion tensor imaging study.ParticipantsThirty-seven AOS subjects and 24 age and sex-matched healthy subjects.Outcome measuresUsing probabilistic tractography, cortical regions with the highest connection probability for each striatal voxel were determined, and correlated with IQ and specific cognitive functions after co-varying for age and sex. Fractional anisotropy (FA) from individual tracts was a secondary measure.ResultsBayesian Structural Equation modeling of FA from 12 frontostriatal tracts showed processing speed to be an intermediary variable for cognition. AOS patients demonstrated generalised cognitive impairment with specific deficits in verbal learning and memory and in processing speed after correction for IQ. Dorsolateral prefrontal cortex connectivity with the striatum correlated positively with these measures and with IQ. DTI voxel-wise comparisons showed lower connectivity between striatum and the motor and lateral orbitofrontal cortices bilaterally, the left amygdalohippocampal complex, right anterior cingulate cortex, left medial orbitofrontal cortex and right dorsolateral prefrontal cortex.ConclusionsFrontostriatal dysconnectivity in large WM tracts that can explain core cognitive deficits are evident during adolescence. Processing speed, which is affected by alterations in WM connectivity, appears an intermediary variable in the cognitive deficits seen in schizophrenia.
Structural alterations of the superior temporal gyrus in schizophrenia: Detailed subregional differences
Authors: Ohi K. , Matsuda Y. , Shimada T. , Yasuyama T. , Oshima K. , Sawai K. , Kihara H. , Nitta Y. , Okubo H. , Uehara T. , Kawasaki Y. 
Published: April 2016

AbstractBackgroundReduced gray matter volumes in the superior temporal gyrus (STG) have been reported in patients with schizophrenia. Such volumetric abnormalities might denote alterations in cortical thickness, surface area, local gyrification or all of these factors. The STG can be anatomically divided into five subregions using automatic parcellation in FreeSurfer: lateral aspect of the STG, anterior transverse temporal gyrus of Heschl gyrus (HG), planum polare (PP) of the STG, planum temporale (PT) of the STG and transverse temporal sulcus.MethodsWe acquired magnetic resonance imaging (MRI) 3T scans from 40 age- and sex-matched patients with schizophrenia and 40 healthy subjects, and the scans were automatically processed using FreeSurfer. General linear models were used to assess group differences in regional volumes and detailed thickness, surface area and local gyrification.ResultsAs expected, patients with schizophrenia had significantly smaller bilateral STG volumes than healthy subjects. Of the five subregions in the STG, patients with schizophrenia showed significantly and marginally reduced volumes in the lateral aspect of the STG and PT of the STG bilaterally compared with healthy subjects. The volumetric alteration in bilateral lateral STG was derived from both the cortical thickness and surface area but not local gyrification. There was no significant laterality of the alteration in the lateral STG between patients and controls and no correlation among the structures and clinical characteristics.ConclusionsThese findings suggest that of five anatomical subregions in the STG, the lateral STG is one of the most meaningful regions for brain pathophysiology in schizophrenia.
Leptin in bipolar disorder: A systematic review and meta-analysis
Authors: Fernandes B.S. , Dash S. , Jacka F. , Dodd S. , Carvalho A.F. , Köhler C.A. , Steiner J. , da Graça Cantarelli M , Nardin P. , Gonçalves C.-A. , Berk M. 
Published: April 2016

AbstractBackgroundBipolar disorder (BD) is a psychiatric disorder associated with increased rates of obesity and inflammation. Leptin is an adipokine that is mainly produced by the white adipose tissue in response to insulin. It stimulates the immune system, increasing the production of pro-inflammatory cytokines. There is currently uncertainty regarding possible alterations in peripheral leptin levels across the mood states in BD.MethodsThis study comprises a between-group meta-analysis comparing serum and plasma leptin levels in people with BD in mania, depression or euthymia and healthy controls. We conducted a systematic search for all possibly eligible-English and non-English peer-reviewed articles. We calculated the effect size (ES) utilizing Hedges’ adjusted g using random effects.ResultsEleven studies were included in the meta-analyses, providing data on 1118 participants. Serum and plasma leptin levels were not altered in subjects with BD when compared to healthy controls in mania (g = −0.99, 95% CI −2.43 to 0.43, P = 0.171), in depression (g = 0.17, 95% CI −0.45 to 0.79, P = 0.584), or in euthymia (g = 0.03, 95% CI −0.39 to 0.46, P = 0.882). However, we did observe a stronger association between leptin levels and both age and BMI in patients with BD in euthymia compared to healthy controls, such that the greater the age of the individuals, the greater the difference in leptin levels between BD and controls; and the higher the BMI, the greater the difference in leptin levels between BD and controls.ConclusionsOur meta-analysis provides evidence that leptin levels are not altered in BD across the mood spectrum compared to healthy controls. The disproportionate increase of leptin levels with increase in BMI in BD speaks in favour of a potential inflammatory role of white adipose tissue in BD and a disproportionate increase of leptin levels with increase in age.
Tratamiento de la disociación estática escafolunar utilizando una plastia de palmar mayor: resultados preliminares
Authors: Gómez M. , Frutos F. , Carrascosa P. , Lluch R. 
Published: April 2016

Objetivo: Examinar los primeros resultados de la reconstrucción de la zona dorsal y palmar del ligamento escafolunar utilizando una plastia de palmar mayor en el tratamiento de la inestabilidad escafolunar estática reductible.Material y método: Desde junio del 2012, 7 pacientes con inestabilidad escafolunar estática reductible fueron intervenidos con reconstrucción de la zona dorsal y palmar del ligamento escafolunar utilizando una plastia de palmar mayor. La edad media de los pacientes fue de 44años. El seguimiento medio fue de 18 meses (rango 12-35 meses).Resultados: Los resultados funcionales según Green y O’Brien fueron buenos en 6 pacientes y satisfactorios en el otro y según las escalas Quick-DASH y Mayo modificada, 6,3 y 80 respectivamente. Radiográficamente, la diastasis escafolunar y el ángulo escafolunar preoperatorio pasaron de 4,6 a 2,4 mm y de 59,3 a 46,6°, respectivamente, al final del seguimiento.Conclusiones: Los resultados clínicos y radiográficos al final del seguimiento son buenos. Esta técnica permite reconstruir la zona dorsal y palmar del ligamento interóseo escafolunar y reforzar y tensar los ligamentos escafo-trapecio-trapezoidales reconstruyendo eficazmente la anatomía del carpo. Si bien los resultados son esperanzadores, se necesita un mayor número de pacientes y mayor seguimiento para obtener conclusiones más definitivas.
Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel
Authors: Tarnopolsky Mark , Katzberg Hans , Petrof Basil J. , Sirrs Sandra , Sarnat Harvey B. , Myers Kimberley , Dupré Nicolas , Dodig Dubravka , Genge Angela , Venance Shannon L. , Korngut Lawrence , Raiman Julian , Khan Aneal 
Published: April 2016

AbstractPompe disease is a lysosomal storage disorder caused by a deficiency of the enzyme acid alpha-glucosidase. Patients have skeletal muscle and respiratory weakness with or without cardiomyopathy. The objective of our review was to systematically evaluate the quality of evidence from the literature to formulate evidence-based guidelines for the diagnosis and management of patients with Pompe disease. The literature review was conducted using published literature, clinical trials, cohort studies and systematic reviews. Cardinal treatment decisions produced seven management guidelines and were assigned a GRADE classification based on the quality of evidence in the published literature. In addition, six recommendations were made based on best clinical practices but with insufficient data to form a guideline. Studying outcomes in rare diseases is challenging due to the small number of patients, but this is in particular the reason why we believe that informed treatment decisions need to consider the quality of the evidence.