Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What is your approach to systemic treatment of a rapidly progressing, symptomatic, unresectable MPNST arising from a plexiform neurofibroma in a patient with NF1?
MPNST is a different ballgame than plexiform neurofibroma. Unfortunately, it is not chemotherapy-sensitive. We currently do not have a good option for MPNST. Perhaps clinical trial enrollment works best. We also do NGS to look for targets, apart from NF1. MEK inhibitors have been tried in the past w...
How would you approach the prevention of irinotecan-induced diarrhea in a patient who previously experienced a diffuse rash with cefixime?
Prevention of irinotecan-induced diarrhea commonly uses prophylactic oral cephalosporins such as cefixime or cefpodoxime, which suppress intestinal β-glucuronidase–producing bacteria that reactivate SN-38 glucuronide and drive delayed diarrhea [1]. In a patient with a prior diffuse rash to cefixime,...
For what patients/diagnoses do you currently use sodium thiosulfate to reduce the risk of cisplatin associated ototoxicity?
Sodium thiosulfate (STS) is approved in the US (FDA) and Europe (EMA) for the prevention of cisplatin-induced hearing loss (CIHL) in patients 1 month and older treated with cisplatin for any type of cancer. Although intravenous STS has not been subjected to a randomized clinical trial for patients >...
What is your approach to patients/parents seeking alternative or complementary treatment in pediatric oncology patients?
Establishing an open dialogue and collaborative partnership with patients and caregivers regarding complementary and alternative therapies is essential.A critical first step is to clearly distinguish between complementary and alternative medicine. These terms are frequently used interchangeably, yet...
For patients with microcytosis MCV 75-79 and normal Hb, low TIBC, and normal ferritin do you always rule out thalassemia?
Microcytosis is typical in thalassemia. With a normal ferritin and hemoglobin concentration, I would start screening by measuring HPLC, HbA2 levels that are high in beta-thalassemia carriers. (HbA2 can be normal with “mild” thalassemia alleles and for several other reasons.) Microcytosis without iro...
What is the target ferritin level for patients with hereditary hemochromatosis and signs of end-organ damage?
I believe the best marker to guide phlebotomy therapy for iron overload is the serum ferritin concentration. I use a target ferritin level of approximately 50 ng/ml. However, one could justify a ferritin level of <200 ng/ml from the literature of serum ferritin compared to body iron stores in HFE he...
How do you approach the treatment of teen patients with recurrent mediastinal germ cell tumor who have failed standard therapy, radiation, and HD therapy/stem cell rescue?
The query failed to delineate whether this was seminoma or non-seminoma, but probably does not matter in this advanced stage of a heavily treated patient. Also failed to address the anatomical location of active cancer. Whether this is a teen or older patient is irrelevant. For mediastinal seminoma,...
Do you routinely consider FDG PET/CT imaging for workup of fever of unknown origin?
The landscape of FUO and IUO and our clinical approach to diagnosing its cause has changed significantly over the past several decades. More sensitive microbiologic screening for infectious etiologies, including syndromic molecular panels and next-generation sequencing are now clinically available a...
Can a patient still have primary HLH even in the absence of any HLH associated genetic mutations?
Yes, a patient can still have primary Hemophagocytic Lymphohistiocytosis (HLH) even in the absence of identified HLH-associated genetic mutations.Primary HLH, also known as familial HLH, is typically linked to mutations in genes related to the immune system, such as PRF1, UNC13D, STX11, STXBP2, and ...
Can lupus anticoagulant be positive despite a normal aPTT?
aPTT is one of the assays that may be abnormal in the presence of lupus anticoagulant, but not always. Usually, when screening for lupus anticoagulant, there will be a "special" aPTT assay used that is a bit more sensitive to detect lupus anticoagulant. There are several different aPTT-based assays ...