Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
When would you consider upfront eculizumab for sickle cell hyperhemolysis?
What is the optimal age at which a patient with sickle cell disease should undergo allogeneic stem cell transplant?
Ideally, would be at diagnosis to limit sickle related injury and generally, younger patients have less complications but since curative treatment is not perfect, it depends mainly on the donor. For matched sibling donor transplant, under age 5yrs has best outcomes with over 15yrs having more GVHD a...
For patients with incidental findings of venous thromboembolism during workup of a treatable malignancy, how do you approach discontinuation after the treatment is complete?
Your approach to incidental thromboembolism found on the workup of malignancy should be similar to any evaluation of a thrombosis. It should be a structured approach with the following questions evaluated before deciding on long-term or short-term anticoagulation. First, one should determine the loc...
What is your treatment approach for a pediatric patient with uterine embryonal rhabdomyosarcoma found after removal of a prolapsing uterine mass, with no evidence of distant metastasis on imaging?
Based on the location of the uterus, this would be a favorable site. In the scenario presented, the tumor is noted to be removed, but the margin status is not noted, which could greatly affect the approach. Also, the exact uterine location (such as cervix vs body of the uterus) may make a difference...
How do you treat refractory cold agglutinin disease?
If the patient has refractory cold agglutinin disease not associated with malignant lymphoproliferative disease, I have used intermediate dose Cytoxan. However, there is recent evidence that inhibition of C1s and thus complement activation by a monoclonal antibody sutimlimab markedly reduces hemolys...
Is it true that a ferritin above 200 essentially rules out iron deficiency?
No, I do not think that a ferritin >200 ug/L essentially "rules out" iron deficiency. Ferritin is an acute phase reactant and can be elevated in myriad conditions including kidney disease, autoimmune disorders, etc. The transferrin saturation (measure of serum iron/TIBC) is an important marker of ir...
Are there instances where you would use C5 complement inhibitors for isolated elevation of sc5b-9 in the absence of other TA-TMA features?
A recent prospective open-label prospective trial, enrolling 21 patients, by Jodele et al., PMID 37946262, published in Blood March 2024, used elevation in sc5b-9 as well as proteinuria as eligibility for the start of therapy with a C5 blocker. They suggest that early treatment of TA-TMA may be of b...
How would you optimally manage a patient with a MDS/MPN overlap syndrome who has both transfusion-dependent anemia and marked thrombocytosis?
Patients with MDS/MPN marked by severe anemia and thrombocytosis likely have the MDS with ring sideroblast with thrombocytosis (MDS-RS-T) subtype and have a high frequency of SF3B1 and JAK2 mutations. Clinically, they resemble a fusion of MDS-RS and essential thrombocythemia (ET), both of which tend...
What is your threshold for pursuing bone marrow biopsy in a lupus patient with cytopenias?
I rarely do bone marrow biopsies in patients with SLE as cytopenias are caused primarily by the disease itself and/or medications, primarily Azathioprine and Cyclophosphamide. SLE patients routinely have profound lymphopenia, neutropenia, and thrombocytopenia. I consider bone marrow biopsy in patien...
In a patient with sickle cell disease on hydroxyurea who is planning to have a family, for how long should they be off the drug before trying to conceive?
There is some consensus that pregnant women should not take hydroxyurea and should stop taking this when found to be pregnant. (Smith-Whitley, PMID 25472967) As far as I know, there is no data on how long hydroxyurea should be discontinued in either men or women before attempting pregnancy. In fact,...