Ticagrelor vs placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: the HESTIA3 study.

The phase 3 HESTIA3 study assessed the efficacy and safety of the reversible P2Y12 inhibitor ticagrelor vs placebo in preventing vaso-occlusive crises in pediatric patients with sickle cell disease (SCD). Patients aged 2 to 17 years were randomly assigned 1:1 to receive weight-based doses of ticagrelor or matching placebo. The primary end point was the rate of vaso-occlusive crises, a composite of painful crises and/or acute chest syndrome (ACS). Key secondary end points included number and duration of painful crises, number of ACS events, and number of vaso-occlusive crises requiring hospitalization or emergency department visits. Exploratory end points included the effect of ticagrelor on platelet activation. In total, 193 patients (ticagrelor, n = 101; placebo, n = 92) underwent randomization at 53 sites across 16 countries. The study was terminated 4 months before planned completion for lack of efficacy. Median ticagrelor exposure duration was 296.5 days. The primary end point was not met: estimated yearly incidence of vaso-occlusive crises was 2.74 in the ticagrelor group and 2.60 in the placebo group (rate ratio, 1.06; 95% confidence interval, 0.75-1.50; P = .7597). There was no evidence of efficacy for ticagrelor vs placebo across secondary end points. Median platelet inhibition with ticagrelor at 6 months was 34.9% predose and 55.7% at 2 hours' postdose. Nine patients (9%) in the ticagrelor group and eight patients (9%) in the placebo group had at least one bleeding event. In conclusion, no reduction of vaso-occlusive crises was seen with ticagrelor vs placebo in these pediatric patients with SCD. This trial was registered at www.clinicaltrials.gov as #NCT03615924.

Glioma Presenting as an Isolated Facial Nerve Palsy: A Case Report.

A peripheral palsy of the facial nerve that results in muscle weakness on one side of the face usually manifests as Bell's palsy. Glioma in the left half of the pons and middle cerebellar peduncle is a rare cause of isolated infranuclear facial paralysis. We report a case of 12- year-old female patient who came to our hospital with isolated unilateral facial palsy but turned out to have a low grade glioma.

Is it reliable to measure the forearm blood pressure in children?

BACKGROUND: When the upper arm (UA) is inaccessible or a standard-sized blood pressure (BP) cuff is unavailable, some healthcare workers use the forearm (FA) to measure BP with a mercury sphygmomanometer. OBJECTIVE: The objective was to determine the accuracy of BP measurement in the arm and FA. DESIGN: Prospective, randomized study. SETTING: Department of Pediatrics, JNMC, Sawangi (Meghe). PARTICIPANTS: A total of 72 children aged 5-15 years. MEASUREMENTS: Mercury and Automatic (OMRON Tokyo, 108-0075 Japan) BP measurements were recorded from the arm and FA at 2 min intervals. RESULTS: In our study, 72 children of both sexes were enrolled. The mean age of the children was 10.13 ± 2.82 years, and 48% were females. Pearson's correlation coefficient between FA and UA systolic BP (SBP) measured by mercury was 0.782, and for diastolic BP (DBP) it was 0.824. Similarly, Pearson's correlation coefficient between FA and UA SBP measured with an automated device (OMRON) was 0.843, and for DBP it was 0.846. The average readings for the SBP and DBP were higher in the FA than in the UA by approximately 3 mmHg. There was a statistically significant difference in both SBP and DBP. CONCLUSIONS: The FA is an acceptable method of BP monitoring when the UA cannot be accessed. The pressure from FA is probably higher than it would be from UA.

Uhl's Anomaly with Absent Tricuspid Valve in an Infant.

Uhl's anomaly is characterized by absence of the myocardial layer of the right ventricle, with opposition of the endocardium and epicardium. It is rarely associated with other congenital malformation. Here, we reported the rare association of Uhl's anomaly with absent tricuspid valve in an infant.

Unicuspid Aortic Valve in Infant.

The unicuspid aortic valve (UAV) is a very rare congenital anomaly, which usually presents as aortic stenosis, incompetence, or a combination of both. Here, we present a case of UAV with moderate aortic stenosis detected by transthoracic echocardiography in the infant.

A case report of ectopia cordis and omphalocele.

A rare congenital defect in fusion of the anterior chest wall resulting in an extrathoracic location of the heart. Cantrell's pentalogy is a congenital anomaly resulting from embryologic development defect and consists of the following: A deficiency of the anterior diaphragm, a midline supraumbilical abdominal wall defect, a defect in the diaphragmatic pericardium, congenital intracardiac abnormalities, and a defect of the lower sternum. Here we report a rare case of ectopic cordis with omphalocele.

Unusual manifestation of Marden-Walker syndrome.

Marden-Walker syndrome (MWS) is characterized by multiple joint contractures, a mask-like face with blepharophimosis, micrognathia, high-arched or cleft palate, low-set ears, decreased muscular bulk, arachnodactyly, and kyphoscoliosis. We report a case of MWS along with unusual manifestation of neurological, cardiovascular, and genitourinary system.

Neonatal genital prolapse.

Neonatal genital prolapse is a rare condition, usually associated with congenital spinal defects and is often resistant to simple reduction. A case of complete uterine prolapse which was noted shortly after birth in a female baby with a meningocele at the lumbar region with rectal prolapse is reported. The vagina and uterus was restored to their normal position but came out immediately after reduction.

Renal abscess in a child with sickle cell anemia.

Renal abscess is rare in children and the usual presenting features include fever, lumbar pain, abdominal pain and occasional flank mass. Renal ultrasonography facilitates an early diagnosis and helps in percutaneous drainage. We herewith report on a child with sickle cell anemia who developed a renal abscess.

Pain response of neonates to venipuncture.

OBJECTIVE: To study the physiological and behavioral response to pain. METHODS: 80 healthy neonates requiring bilirubin estimation, blood sugar etc, were randomly assigned to receive a venous puncture. All parameters were recorded 10 minutes prior, during and 10 minutes after the procedure. Evaluated NIPS score and RR. HR, NIBP and O2 saturation observed on Datex-Ohmeda multimonitor. RESULTS: After the venepuncture, heart rate (p<0.001) and blood pressure (p<0.001) were significantly increased in both the groups but more significant increase was present in Group I (>2.5 kg) as compared to Group II (>2.5 kg). Respiratory rate was also increased but more significant in Group II (p<0.001) whereas oxygen saturation was decreased in both the groups but more significant in Group I (p<0.001). Median Neonatal Infant Pain Scale (NIPS) score was higher in both the Groups (p<0.001). CONCLUSION: The outcome measures appear to be reliable indices of term neonates responses to painful stimulation. NIPS are suitable instruments for neonatal pain evaluation.