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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 | UTC

This 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):

Marie Scully et al. (2024). Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura. N Engl J Med, 390(17), 1584-1596. DOI: 10.1056/NEJMoa2314793.

 


Global prevalence, years lived with disability, and trends in anemia burden by severity and cause, 1990–2021

3 Aug, 2023 | 13:43h | UTC

Prevalence, years lived with disability, and trends in anaemia burden by severity and cause, 1990–2021: findings from the Global Burden of Disease Study 2021 – The Lancet Haematology

News Release: New study reveals global anemia cases remain persistently high among women and children. Anemia rates decline for men – Institute for Health Metrics and Evaluation

 

Commentary on Twitter

 


Practice Guidance | Management of febrile neutropenia in immunocompetent children and youth

3 Aug, 2023 | 13:38h | UTC

Management of febrile neutropenia in immunocompetent children and youth – Canadian Paediatric Society

 


RCT | Thrice-weekly oral iron shows non-inferiority and fewer gastrointestinal effects in treating iron-deficiency anemia

20 Jun, 2023 | 12:48h | UTC

A 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

Significant haemoglobinopathies: A guideline for screening and diagnosis: A British Society for Haematology Guideline – British Journal of Haematology

 


Current guidelines for perioperative management and transfusion decisions in sickle cell patients

30 May, 2023 | 11:57h | UTC

Current Evidence and Rationale to Guide Perioperative Management, Including Transfusion Decisions, in Patients With Sickle Cell Disease – Anesthesia & Analgesia

 


RCT | Hydroxyurea for secondary stroke prevention in children with sickle cell anemia

17 Apr, 2023 | 12:48h | UTC

Hydroxyurea for secondary stroke prevention in children with sickle cell anemia in Nigeria: a randomized controlled trial – Blood

 


Guidelines on diagnosis and management of chronic neutropenia in adults and children

10 Apr, 2023 | 13:25h | UTC

The European Guidelines on Diagnosis and Management of Neutropenia in Adults and Children: A Consensus Between the European Hematology Association and the EuNet-INNOCHRON COST Action – HemaSphere

 

Commentary on Twitter

 


Hydroxyurea use in youths with sickle cell anemia: underused despite updated 2014 guidelines

5 Apr, 2023 | 13:16h | UTC

Changes in Hydroxyurea Use Among Youths Enrolled in Medicaid With Sickle Cell Anemia After 2014 Revision of Clinical Guidelines – JAMA Network Open

Original Guideline: National Heart, Lung, and Blood Institute. Evidence-based management of sickle cell disease: expert panel report, 2014

 

Commentary on Twitter

 


RCTs | Fitusiran prophylaxis reduces bleeding in people with hemophilia A or hemophilia B

3 Apr, 2023 | 13:44h | UTC

Efficacy 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 | UTC

Summary:

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.

Article: Oral iron supplementation and anaemia in children according to schedule, duration, dose and cosupplementation: a systematic review and meta-analysis of 129 randomised trials – BMJ Global Health

 


RCT | 2-y outcomes confirm the harms caused by high prophylactic platelet transfusion thresholds in preterm infants

28 Feb, 2023 | 13:53h | UTC

Summary: 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.

Article: Two-year outcomes following a randomised platelet transfusion trial in preterm infants – ADC Fetal & Neonatal

Original Study: Randomized Trial: Platelet-Transfusion Thresholds in Neonates

 

Commentary from the author on Twitter (thread – click for more)

 


Consensus Paper | Pediatric traumatic hemorrhagic shock

17 Feb, 2023 | 13:15h | UTC

Pediatric traumatic hemorrhagic shock consensus conference recommendations – Journal of Trauma and Acute Care Surgery

Preface: 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 | UTC

Efanesoctocog 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

 


RCT | IV ferric carboxymaltose vs. oral ferrous fumarate in anemic children with inflammatory bowel disease

30 Jan, 2023 | 00:18h | UTC

Ferric carboxymaltose versus ferrous fumarate in anemic children with inflammatory bowel disease: the POPEYE randomized controlled clinical trial – The Journal of Pediatrics

 


Preliminary study suggests that Edoxaban is a safe alternative for preventing thromboembolism in pediatric patients with cardiac disease.

9 Dec, 2022 | 13:07h | UTC

Edoxaban for Thromboembolism Prevention in Pediatric Patients With Cardiac Disease – Journal of the American College of Cardiology (link to abstract – $ for full-text)

Editorial: Advancing Anticoagulation for Children With Cardiac Disease – Journal of the American College of Cardiology

Commentary: Edoxaban for Prevention of Blood Vessels Being Blocked by Clots (Thrombotic Events) in Children at Risk Because of Cardiac Disease – ENNOBLE-ATE – American College of Cardiology

Related: [Abstract Only] Randomized Trial: Direct Oral Anticoagulant Similarly Effective as Standard Anticoagulants for the Treatment of Acute Venous Thromboembolism in Children

 


RCT | Leniolisib for Activated phosphoinositide 3-kinase delta syndrome.

22 Nov, 2022 | 13:12h | UTC

Randomized, Placebo-Controlled, Phase 3 Trial of PI3Kδ Inhibitor Leniolisib for Activated PI3Kδ Syndrome – Blood 

 


Review | Thrombotic microangiopathy in children.

5 Aug, 2022 | 13:56h | UTC

Thrombotic 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

Incidence of Acute Chest Syndrome in Children With Sickle Cell Disease Following Implementation of the 13-Valent Pneumococcal Conjugate Vaccine in France – JAMA Network Open

 

Commentary on Twitter

 


Guideline | Role of IVIG in the management of Rh- and ABO-mediated hemolytic disease of the newborn.

2 Aug, 2022 | 12:18h | UTC

International guidelines regarding the role of IVIG in the management of Rh- and ABO-mediated haemolytic disease of the newborn – British Journal of Haematology

 


RCT | Ticagrelor is ineffective for reducing vaso-occlusive crises in pediatric sickle cell disease.

25 Jul, 2022 | 12:50h | UTC

Ticagrelor versus placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: the HESTIA3 study – Blood

 


Review | Sickle cell disease.

7 Jul, 2022 | 12:59h | UTC

Sickle 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

Egyptian evidence-based consensus on clinical practice guidelines for the diagnosis and treat-to-target management of macrophage activation syndrome in children – Egyptian Rheumatology and Rehabilitation

 


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