Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What additional testing is recommended with heterozygous HFE C282Y mutation whose ferritin is elevated and transferrin saturation percentage is at baseline?
Here, both serum ferritin and transferrin saturation are significantly elevated. Assuming the patient is asymptomatic and LFTs are normal, this profile is suggestive of iron overload. I assume Hb is normal? Would do MRI to look for hepatic iron overload.
Is there any indication for monitoring for renal medullary carcinoma in patients with sickle cell trait?
Hematuria occurs episodically in approximately 5% of people with sickle cell trait. Although often recurrent and sometimes producing anemia, the causes of hematuria are usually benign and it is self-limited. Medullary carcinoma of the kidney is a rare highly aggressive tumor that occurs almost exclu...
For mild to moderate hemophilia B, do you routinely screen for inhibitors?
Most experts recommend testing for an inhibitor within 6 to 12 months after concentrate therapy, before any major surgery, and if the patient has a poor response to concentrate therapy (lower than the expected level achieved, or shortened half-life). An annual test is also recommended. WFH Guideline...
How do you approach an ITP patient who responds very well to steroids but the response is short-lived?
Introduce second line therapy. Rituxan pr TPO-RA.
How do you manage significant asymptomatic indirect hyperbilirubinemia in patients with hemolytic diseases (SCD, HS, etc.)?
Hello, Indirect bilirubin is from hemolysis, not cholestasis (a few exceptions from rare syndromes), so not associated with stones, and would not expect ursodiol to help. It's a marker of how bad the sickling is - along with LDH, reticulocyte count, and MCHC (which measures cell density, i.e., irrev...
What are the treatment options for relapsed T-ALL in a patient who was nonadherent with AALL and hyper-CVAD regimens?
Regimens to consider with data for R/R T-ALL include: Nelarabine +/- chemotherapy. There are retrospective data to support adding nelarabine to a chemo backbone (Shimony et al., PMID 36508268 and Shimony et al., PMID 37389830). HMA + venetoclax (Pinton et al., PMID 37889114 and Cao et al., Blood 202...
How would you treat an advanced stage small-cell carcinoma of the ovary, hypercalcemic type? (SCCOHT)?
Small cell carcinoma of the ovary hypercalcemic type (SCCOHT) is an exceptionally rare tumor affecting patients from infancy to at least the fifth decade of life. SCCOHT tumors are characterized by mutations of the SWI/SNF member SMARCA4 that encodes BRG1. Given the rarity of SCCOHT, limited prospec...
How quickly do you expect iron stores to decrease after starting iron chelators?
This is a complex answer to what seems like a simple question. It is not a standard rate of decline because each patient's situation is different. It is always about the balance of how much iron is going in vs. how much iron is being excreted. Essentially there are 4 variables that must be considere...
How would you approach secondary stroke prevention in an adult with Hemoglobin SC disease?
Stroke is less common in HbSC disease than it is in HbS homozygotes (Ohene-Frempong et al., PMID 9414296). Thus, there are no studies focused on primary or secondary stroke prevention in HbSC disease. Recent guidelines for stroke management were “silent” on stroke in HbSC disease (DeBaun et al., PMI...
How do you approach IVIG replacement for pediatric patients with low IgG during treatment for hematologic malignancies?
We monitor IgG levels at the beginning of each chemotherapy cycle for infants, for patients with Down syndrome, for those receiving blinatumomab, and for patients who are hypogammaglobulinemic with recurrent bacterial infections, and we replace when IgG levels are <400 mg/DL for down syndrome and <5...