Pediatrics – Hematology
Randomized Crossover Trial: Prophylactic recombinant ADAMTS13 prevents acute events in congenital thrombotic thrombocytopenic purpura – N Engl J Med
6 May, 2024 | 06:22h | UTCThis study evaluates the efficacy and safety of recombinant ADAMTS13 compared to standard plasma-derived therapy in managing congenital thrombotic thrombocytopenic purpura (TTP). In a phase 3, open-label, crossover trial involving 48 patients, each participant underwent two 6-month prophylaxis periods, receiving either recombinant ADAMTS13 or standard therapy, followed by a switch to the alternate treatment. Results indicate that recombinant ADAMTS13 prevented acute TTP events during prophylaxis, with no events recorded, versus one event under standard therapy. Furthermore, recombinant ADAMTS13 was associated with significantly lower rates of thrombocytopenia and adverse events compared to standard therapy. The treatment increased ADAMTS13 activity to approximately 100% of normal levels, with no development of neutralizing antibodies. Overall, recombinant ADAMTS13 was found to be safe and more effective than standard therapy in preventing TTP events and manifestations.
Reference (link to abstract – $ for full-text):
Global prevalence, years lived with disability, and trends in anemia burden by severity and cause, 1990–2021
3 Aug, 2023 | 13:43h | UTC
Commentary on Twitter
NEW #GBD study by @IHME_UW on the #anaemia burden by severity and cause, 1990–2021?https://t.co/NbiEDSkbGI
Despite a decrease in prevalence, the number of people with anaemia increased globally
In every region females had a higher #anemia YLDs than males pic.twitter.com/oFx4vHRhs1
— The Lancet Haematology (@TheLancetHaem) August 1, 2023
Practice Guidance | Management of febrile neutropenia in immunocompetent children and youth
3 Aug, 2023 | 13:38h | UTC
RCT | Thrice-weekly oral iron shows non-inferiority and fewer gastrointestinal effects in treating iron-deficiency anemia
20 Jun, 2023 | 12:48h | UTCA randomized controlled trial of thrice-weekly versus thrice-daily oral ferrous fumarate treatment in adult patients with iron-deficiency anemia – Ann Hematol (if the link is paywalled, try this one)
Guideline | Screening and diagnosis of significant hemoglobinopathies
20 Jun, 2023 | 12:32h | UTC
Current guidelines for perioperative management and transfusion decisions in sickle cell patients
30 May, 2023 | 11:57h | UTC
RCT | Hydroxyurea for secondary stroke prevention in children with sickle cell anemia
17 Apr, 2023 | 12:48h | UTC
Guidelines on diagnosis and management of chronic neutropenia in adults and children
10 Apr, 2023 | 13:25h | UTC
Commentary on Twitter
Neutropenia diagnosis & management were previously based on physicians' experience or local practices. To harmonize #hematology treatment, #EHA & @eunet_innochron have published a new #EHAGuideline on #neutropenia in adults & children in @Hemasphere_EHA: https://t.co/ifYEtzNlAv pic.twitter.com/CnKuqG03Up
— European Hematology Association (@EHA_Hematology) April 3, 2023
Hydroxyurea use in youths with sickle cell anemia: underused despite updated 2014 guidelines
5 Apr, 2023 | 13:16h | UTCOriginal Guideline: National Heart, Lung, and Blood Institute. Evidence-based management of sickle cell disease: expert panel report, 2014
Commentary on Twitter
Hydroxyurea prescribing in youths with sickle cell anemia insured by Medicaid modestly after the @nih_nhlbi guidelines recommended hydroxurea in 2014. Increasing hydroxyurea use may require a multifaceted approach. https://t.co/tkY0HiMvkv
— JAMA Network Open (@JAMANetworkOpen) March 24, 2023
RCTs | Fitusiran prophylaxis reduces bleeding in people with hemophilia A or hemophilia B
3 Apr, 2023 | 13:44h | UTCEfficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial – The Lancet (link to abstract – $ for full-text)
Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trial – The Lancet Haematology (link to abstract – $ for full-text)
News Release: Monthly injections of fitusiran reduces bleeds in patients with haemophilia A and B – The Lancet
M-A | Oral iron supplementation and anemia in children according to schedule, duration, dose and cosupplementation
7 Mar, 2023 | 12:53h | UTCSummary:
The article is a systematic review and meta-analysis of 129 randomized trials on iron supplementation and anemia in children. The study aimed to identify the optimal schedule, duration, dose, and cosupplementation regimen for iron supplementation in children and adolescents aged under 20 years.
The results showed that frequent (3-7 times/week) and intermittent (1-2 times/week) iron supplementation could be equally effective at increasing hemoglobin and decreasing anemia, iron deficiency, and iron deficiency anemia.
The study supports WHO recommendations regarding the frequency, duration, and dose of iron supplementation, including 3 months annually of daily oral iron supplementation for children aged 6 months to 12 years living in regions with a high burden of anemia. However, it also suggests that weekly iron supplementation might be considered an alternative to the recommended daily regimen in some contexts, given evidence of similar efficacy.
RCT | 2-y outcomes confirm the harms caused by high prophylactic platelet transfusion thresholds in preterm infants
28 Feb, 2023 | 13:53h | UTCSummary: The study investigated the long-term outcomes of preterm infants who participated in a randomized trial of platelet transfusions at different threshold levels. Infants with platelet counts below 50×109/L were enrolled and randomized to either a higher threshold of 50×109/L or a lower threshold of 25×109/L. The study found that infants randomized to the higher threshold had a higher rate of death or significant neurodevelopmental impairment at a corrected age of 2 years compared to the lower threshold group. The study further supports evidence of harm caused by high prophylactic platelet transfusion thresholds in preterm infants. The mechanisms by which platelet transfusions could mediate harmful effects remain unknown, but the study identified increased rates of intraventricular hemorrhage and bronchopulmonary dysplasia in the higher threshold group, which may contribute to the adverse outcomes observed.
Original Study: Randomized Trial: Platelet-Transfusion Thresholds in Neonates
Commentary from the author on Twitter (thread – click for more)
We already knew from the @PlaNeT2_trial that a higher prophylactic platelet transfusion threshold (<50) in preterm babies is associated with significantly increased mortality or major bleeding compared with a lower one (<25)… https://t.co/pJrE44BIIx
— Carmel Moore (@carmelmoore) February 22, 2023
Consensus Paper | Pediatric traumatic hemorrhagic shock
17 Feb, 2023 | 13:15h | UTCPreface: Pediatric traumatic hemorrhagic shock consensus conference
Other articles in the supplement:
Pediatric traumatic hemorrhagic shock consensus conference research priorities
Building an optimal pediatric trauma program
Time is tissue: Barriers to timely transfusion after pediatric injury
Resuscitative practices and the use of low-titer group O whole blood in pediatric trauma
Tranexamic acid in pediatric hemorrhagic trauma
Blood banking considerations in pediatric trauma
Detecting traumatic brain injury–induced coagulopathy: What we are testing and what we are not
RCT | Effects of once-weekly prophylaxis with Efanesoctocog Alfa in patients with severe Hemophilia A
30 Jan, 2023 | 01:14h | UTCEfanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A – New England Journal of Medicine (link to abstract – $ for full-text)
Commentary: Once-Weekly Efanesoctocog Alfa Beneficial in Severe Hemophilia A – HealthDay
Commentary on Twitter with a video summary
Efanesoctocog alfa is a new class of factor VIII replacement for hemophilia A that has a longer half-life than other factor VIII products, thus allowing once-weekly dosing. New research findings are summarized in a short video. https://t.co/YuXzifUh8m pic.twitter.com/POCAAfeEni
— NEJM (@NEJM) January 27, 2023
RCT | IV ferric carboxymaltose vs. oral ferrous fumarate in anemic children with inflammatory bowel disease
30 Jan, 2023 | 00:18h | UTC
Preliminary study suggests that Edoxaban is a safe alternative for preventing thromboembolism in pediatric patients with cardiac disease.
9 Dec, 2022 | 13:07h | UTCEdoxaban for Thromboembolism Prevention in Pediatric Patients With Cardiac Disease – Journal of the American College of Cardiology (link to abstract – $ for full-text)
RCT | Leniolisib for Activated phosphoinositide 3-kinase delta syndrome.
22 Nov, 2022 | 13:12h | UTC
Review | Thrombotic microangiopathy in children.
5 Aug, 2022 | 13:56h | UTCThrombotic microangiopathy in children – Pediatric Nephrology (if the link is paywalled, try this one)
Cohort Study | Incidence of acute chest syndrome in children with sickle cell disease following implementation of the 13-valent pneumococcal conjugate vaccine.
3 Aug, 2022 | 13:53h | UTC
Commentary on Twitter
#OrigInvestigation: Reduction of acute chest syndrome incidence in children with sickle-cell disease following 13-valent pneumococcal conjugate vaccine (PCV13) implementation in France. https://t.co/MuG6T0g5FU
— JAMA Network Open (@JAMANetworkOpen) August 2, 2022
Guideline | Role of IVIG in the management of Rh- and ABO-mediated hemolytic disease of the newborn.
2 Aug, 2022 | 12:18h | UTC
RCT | Ticagrelor is ineffective for reducing vaso-occlusive crises in pediatric sickle cell disease.
25 Jul, 2022 | 12:50h | UTC
Review | Sickle cell disease.
7 Jul, 2022 | 12:59h | UTCSickle Cell Disease: A Review – JAMA (free for a limited period)
Audio Clinical Review: Sickle Cell Disease – JAMA
Consensus Paper: Diagnosis and treat-to-target management of macrophage activation syndrome in children.
24 Jun, 2022 | 11:13h | UTC