Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
How would you approach cytopenias 5 weeks after initial dosing in a young patient with MDS treated with Azacitadine as a bridge to transplant?
Bridging with HMA to transplant in MDS patients is a common practice although it did not show improved outcomes. You will definitely have to r/o other potential causes of pancytopenia (i.e., infections, etc). I would repeat a BM A/Bx to make sure that blasts are not increasing (< 20% and preferably ...
What would your next line treatment be for a patient previously treated with repeat resection, craniospinal irradiation, and initial cisplatin/vincristine/CCNU with medulloblastoma?
As you will agree, this is an extremely unfortunate situation. I am assuming this patient has progressed either while on therapy or soon after completion? Depending on his age, a potential option is to enroll him on any available clinical trials. PBTC58 study uses intrathecal omburtumab along with c...
How do you work up a young patient with increasing ferritin and normal TSAT without infectious, inflammatory, or liver disorders?
This is a tough one. If the ferritin is increasing and the TSAT is normal and there is no evidence of hemochromatosis, the ONLY possible explanation is some underlying morbidity, inflammatory, rheumatologic, malignant, or infectious is present. In pediatrics, HLH (hemophagocytic lymphohistiocytosi...
What is the choice of treatment for a teenager with low risk neuroblastoma s/p resection?
According to the Revised Neuroblastoma Risk Classification (Irwin et al., PMID 34319759), age is not a factor in determining whether someone is low risk. So if this child has been determined to have low-risk neuroblastoma, they have/had an L1 tumor, either without MYCN amplification, or with MYCN am...
How do you integrate surgery in treatment of recurrent tenosynovial giant cell tumors (TGCT) when using Pexidartinib?
This is an interesting question, but one that has little (if any) data to guide management. With recurrent TGCT, the patient presumably already had surgery. It is possible that a good response to pexidartinib may reduce the morbidity of a repeat surgery but the risk of additional recurrence would re...
Which chemo regimen, if any, would you use as adjuvant treatment in a young patient with primary malignant thyroid teratoma?
There is no evidence base to guide you here. The term "malignant teratoma" is not particularly helpful here and does not distinguish between benign teratoma, somatic transformation, or active germ cell malignancies. If classic markers AFP and HCG are significantly elevated, that would favor a malign...
What is the treatment of choice for mixed phenotype ALL?
These are difficult to treat. The limited available data suggest that an ALL–like regimen followed by HSCT may be recommended. If no response is achieved, you could try a myeloid-like strategy. For T/Myeloid, combining asparaginase with AML therapy (FA + PEG or Capizzi II) could be an option. The sp...
Do you stop hydroxyurea or switch to an alternative therapy in a male patient with controlled Hb SC disease who is interested in having children?
Part of the reason the patient may have controlled disease could be the hydroxyurea and so stopping that therapy might not be best for the patient's disease. Also, many patients on hydroxyurea can still get their partner pregnant. So, I would only consider stopping/holding it if the patient is havin...
What is your approach to BK hemorrhagic cystitis not responding to cidofovir?
BK hemorrhagic cystitis can range from completely asymptomatic infection (with positive BK viremia and viruria by PCR) or grade 0 to massive macroscopic hematuria requiring instrumentation for clot evacuation and urinary obstruction requiring bilateral nephrostomy tubes for urine diversion (grade 4)...
Which patients with acute promyelocytic leukemia do you consider maintenance after completing consolidation?
Before we address the role of maintenance in APL, several principles are important to note. First, APL is now a highly curable disease with contemporary therapeutic strategies. Second, we now divide patients into low-risk and high-risk based solely on the presenting WBC (< or =10,000/uL vs >10,000/u...