Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What workup do you consider for a developmentally typical child with multiple large café au lait macules but no other signs of neurofibromatosis?
The likelihood that this is NF1 depends somewhat in part on the age of the child in question. In infants and toddlers, cafe-au-lait macules are usually the first and only clinical sign of NF1. Most children with NF1 will develop skin fold freckling in early childhood, and the majority (probably 60-7...
How would you counsel a female to male transgender patient regarding VTE risk with testosterone therapy, who has additional mild-moderate risk factors for thrombosis?
If physiologic concentrations of testosterone are not exceeded and the hematocrit is monitored to avoid a pathologic level of erythrocytosis, the risk for thrombosis from testosterone GAHT does not appear to in excess of the general population. I would refer you to the following two articles that pr...
How would you evaluate a pediatric patient referred to rule out cancer due to an isolated significantly elevated vitamin B12 level?
Do you recommend giving anti-complement therapy to a patient with approximately 20% PNH clones who has severe aplastic anemia but no other risks for thrombosis or hemolysis?
Depends on the age, the extent of hemolysis, dynamics of PNH clone over time, marrow findings and HSCT donor availability, and also if the patient responds to IST. If the patient has poor response to IST, good donor availability, and no severe hemolysis, I would go straight to transplant. If this i...
What therapy would you consider for refractory pediatric HR AML with KMT2Ar to try to induce remission for transplant?
For any case of refractory AML regardless of the cytogenetics, we turn to regimens used in the relapse setting. Nowadays common choices are based on fludarabine/cytarabine (FLA), CPX-351, and/or venetoclax. For this patient, I would focus on FLA and venetoclax-based regimens. To take advantage of th...
How would you counsel a young female patient who refuses to use contraception during radiotherapy?
A woman who refuses contraception needs careful psychological and psychiatric assessment and considerable time at the time of consultation to understand why she is refusing, especially since this response is neither rational nor logical. The practitioner needs to take the time to discuss the patient...
For patients with Stage IIIB or IV HD flowing Bv-AVEPC with initial large mediastinal adenopathy, how can we avoid ISRT?
It is true that on the clinical trial AHOD1331, patients who presented with large mediastinal adenopathy received radiation therapy as did patients who were slow early responders (Deauville 4 or 5 after two cycles of therapy). It is noteworthy that 3-year event-free survival was extremely high for a...
What experience have you had with familial clustering of polycythemia vera?
JAK2 V617F is a somatic mutation that can be acquired as early as in utero based on elegant work by Williams et al., PMID 35058638.There is some data available regarding familial MPN predisposition syndromes and this review very nicely summarizes much of the available data (Lim et al., PMID 39316992...
Would you consider combining tovorafenib and trametinib as dual therapy?
A phase 1/2 study of tovorafenib and the MEK inhibitor selumetinib is planned through the Developmental Therapeutics Committee of the Children's Oncology Group for low-grade gliomas that have failed appropriate prior MAPK blocking therapy and other patients with BRAF/RAF1, RAS, and NF driven maligna...
What is the current opinion on G-CSF usage for AML patients during induction and consolidation phases of treatment?
I agree with you that the use of G-CSF for AML patients is still controversial. There are two reasons for using G-CSF for patients with AML. One is to sensitize, or “prime,” the leukemia by driving quiescent blasts into cell cycle and by mobilizing blasts out of their protective niches and into the ...