Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What treatment approach do you take in a pediatric patient less than 3 years old with malignant papillary epithelioid tumor within the pineal region?
This is an interesting case for a rare tumor, especially in the pediatric population. There is unfortunately no prospective data so clinical management would be drawing from small retrospective series with primarily adult patients. The youngest patient with PTPR in the current literature is 5 years ...
Would you consider salvage SRS in a young patient with locally recurrent ATRT?
Radiotherapy plays an important role in the curative management of ATRT.[Chi et al, JCO 2009; Reddy et al JCO 2020].When relapse occurs, even if it is local, the overall prognosis is often poor. However, I am assuming that this was previously treated, and I would consider re-irradiation with SRS as ...
How would you approach treatment in an otherwise young, fit patient with transformed AML currently in remission with a plan for transplant and was found to have an invasive fungal infection not amenable to resection upon recovery from induction?
This is an all too common problem. Firstly, "not resectable" is sometimes in the eye of the beholder. Ensure all effort is made to resect if this is isolated even if it's a brain lesion or needs a lung resection. Secondly, not all "fungi" are the same. If this is mold, you must be sure it is "not re...
Should regular screening brain MRIs be done for children with germline RB1 mutation in addition to eye exams?
Yes, baseline brain MRI and routine screening approximately every 6 months until the age of 5 years is recommended in any heritable RB patient, including those with bilateral disease or unilateral disease with a family history of the disease (to account for those individuals who may not have an "ide...
How do you approach a patient with metastatic rhabdomyosarcoma complicated by sinusoidal obstruction syndrome (SOS) after neoadjuvant chemotherapy and radiation therapy?
This is a challenging and unfortunate complication of therapy. Based solely on the information provided and with only 3 fractions left, it sounds like the patient received the majority of radiation therapy, and I would forgo the remainder despite the recovery on defibrotide. However, I would conside...
How do you manage leptomeningeal disease in relapsed rhabdomyosarcoma?
I recently had such a case and consulted with a well-known colleague who only specializes in pediatric oncology. The recommendation was to treat with 30Gy in 10 fractions with CSI, which I did. The patient is 5 months post this treatment and is disease-free in the CNS.
How would you treat a young patient with symptomatic Rosai-Dorfman disease who has recurrent, life-threatening pericardial and pleural effusions?
Rosai-Dorfman disease is the most tricky one of the top 3 histiocytosis (ECD, LCH, and RDD). It can behave as benign or as an aggressive disease. Given the rarity of the disease, there are no great prospective studies to guide optimal treatments. First-line treatment includes prednisone or other imm...
Would you give dasatinib to a standard risk pre-B ALL patient with an ABL1 mutation?
I would like to revise my previous answer to this excellent question slightly. While we still do not have solid data to know how to manage such patients, the COG Ph+ B-ALL study (AALL1631) is now open to enrolling Ph-like B-ALL patients with ABL-class fusions regardless of NCI status. Thus, I would ...
How do you treat pediatric AML that has relapsed after transplant?
Until we have more effective therapies, a cure after relapse for a child with AML can only be achieved with stem cell transplant, best done after achieving a second remission. The same is true for relapse after transplant, although in that situation the options may be a bit different. The following ...
How do you manage newly diagnosed stage IV embryonal rhabdomyosarcoma?
Given stage 4 FOX01- RMS, I think you can either use protocol ARST0431 which has 10.8g/m2 (>4y) or 360 mg/kg (<4y) of CPM, or ARST1431 (8.4g/m2) since cumulative CPM dosing is not that much different. I do not dose modify CPM based on the primary site.