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Neuroblastoma

Video Published 2020-05-02 Updated 2026-03-16

Timestops (6)

Introduction to the topic

Introduction to the topic: neuroblastoma management complexities.

Introduction of the experts

Introduction of the experts: Dr. von Allman, Dr. Neumann, Dr. Sandler.

Case presentation begins

Case presentation begins: Prenatal consultation of a 26-week fetus with a left-sided super renal mass.

Discussion on postnatal management

Discussion on postnatal management, including ultrasound and urine caticolamines.

Discussion on the role of MIBG scan and what to look for.

Discussion on observation vs

Discussion on observation vs. surgery after a positive MIBG scan. Mention of Nocktern study.

Topic Overview

A panel discussion on neuroblastoma management featuring three pediatric surgeons: Dr. Dan von Allman (Cincinnati Children's), Dr. Erika Neumann (CS Mott Children's), and Dr. Tony Sandler (Children's National). The discussion covers prenatal diagnosis and observation protocols, staging workup for older children with abdominal masses, and tissue acquisition strategies. Key clinical points include: prenatally diagnosed adrenal masses can be safely observed with serial ultrasound and urine catecholamines through the first year of life with approximately 20% requiring resection; stage MS (formerly 4S) disease with liver involvement requires aggressive observation unless respiratory compromise develops; and for large abdominal masses in older children, complete staging includes cross-sectional imaging, MIBG scan, bone marrow biopsy, and tissue acquisition for pathology and biology studies.

Key Takeaways

Prenatal neuroblastoma can be safely observed with serial US and catecholamines; ~20% need resection, 98% event-free survival. (8:44)
Stage MS with liver mets can be observed unless respiratory compromise develops; then chemo, XRT, or emergent decompression needed. (17:58)
Complete staging for abdominal mass: CT, bone marrow biopsy, MIBG scan, chest CT; 10% of neuroblastomas are MIBG-negative. (23:50)
Open retroperitoneal biopsy preferred over laparoscopic or percutaneous for adequate tissue and bleeding control in large tumors. (27:10)
NMYC amplification in stage MS changes staging to M; biology studies beyond NMYC (ALK, ploidy) require tumor tissue, not just marrow. (20:40)

Inside this episode

Kai, the Library's AI content creator, listened to this episode and mapped who's speaking, the chapters, key claims, and cases. Every item links to the exact moment in the recording.

AI-enriched

Who's speaking

Ray Hanky — host
Todd Ponsky — host
Dan von Allman — guest
Erika Neumann — guest
Tony Sandler — guest

Chapters

0:00Prenatal Diagnosis and Observation — Discussion of differential diagnosis for prenatal suprarenal mass (adrenal hemorrhage vs neuroblastoma), postnatal workup with ultrasound and urine catecholamines, MIBG scanning, and observation protocols based on Nocktern study data showing 20% resection rate with 98% event-free survival.
10:39Surveillance Protocol and Indications for Surgery — Detailed surveillance schedule (intensive first year, then spaced), size criteria for surgery (5 cm threshold), volume increase >50% as trigger, and cautionary case of delayed metastatic presentation at age 3 after initial mass resolution.
15:08Stage MS Disease with Liver Involvement — Management of neuroblastoma with skin lesions and liver metastases (stage MS/4S), respiratory compromise from hepatomegaly as primary concern, treatment options including aggressive observation, chemotherapy, radiation, and emergent decompressive laparotomy, with discussion of biopsy necessity.
21:05Workup of Large Abdominal Mass in Older Child — Initial evaluation with ultrasound and labs, CT scan showing L2 INRG classification tumor with vascular encasement, complete staging workup including MIBG scan, bone marrow biopsy, chest CT, and discussion of PET scan utility (10% of neuroblastomas are MIBG-negative).
27:10Tissue Acquisition Strategies — Debate over biopsy approaches: open retroperitoneal biopsy for adequate tissue for biology studies vs percutaneous vs laparoscopic approaches, with emphasis on obtaining sufficient tissue for NMYC amplification, ALK mutation, and ploidy status beyond what bone marrow alone provides.

Key claims

2:11Adrenal hemorrhage is the most common differential diagnosis for prenatal suprarenal mass, more common with history of fetal stress — Dan von Allman
2:11Other differential diagnoses for suprarenal mass include neuroblastoma, pulmonary sequestration below the diaphragm, and misdiagnosed renal anomaly — Dan von Allman
4:39Familial neuroblastoma occurs in about 1% of patients — Tony Sandler
5:07For prenatal suprarenal mass, first postnatal study should be ultrasound of the abdomen — Todd Ponsky
5:34CT scan or MRI not needed for 3 cm lesion unless urine catecholamines are elevated — Tony Sandler
6:02MIBG scan is the next step if catecholamines are elevated — Dan von Allman
6:02Radiologists are quite good at identifying adrenal hemorrhage on ultrasound — Dan von Allman
8:14In perinatal phase, most common metastatic sites are liver, bone, skin, and lymph nodes — Dan von Allman
8:44Nocktern study data supports observation of prenatal neuroblastoma with careful ultrasound surveillance — Dan von Allman
10:39Of 84 observed patients in Nocktern study, 16 (approximately 20%) underwent resection for growth or family preference — Tony Sandler
11:07Nocktern study showed approximately 98% event-free survival and 100% overall survival in observed prenatal neuroblastoma — Erika Neumann
11:37First-year surveillance protocol: ultrasound and catecholamines at birth, 3 weeks, 6 weeks, 12 weeks, then spaced out to one year — Erika Neumann
11:37After one year, surveillance becomes every six months, then yearly — Erika Neumann
12:27Case report: child with observed prenatal adrenal mass that resolved presented at age 3 with widely metastatic high-risk neuroblastoma — Dan von Allman
14:365 centimeters is used as size cutoff for surgical intervention in observed prenatal masses — Tony Sandler
15:08Volume increase of more than 50% is criterion for considering surgery — Todd Ponsky
15:0850% increase in VMA or HVA prompts consideration of surgery — Todd Ponsky
16:07Laparoscopic approach is reasonable for masses less than 6 centimeters — Erika Neumann
16:36Lymph node status in neuroblastoma is not as important for therapy changes as in Wilms tumor — Dan von Allman
17:00Biology of neuroblastoma is more important than lymph node status for treatment decisions — Erika Neumann
17:30Stage MS (formerly 4S) with skin lesions and liver mets still tends to have good biology — Erika Neumann
17:58Primary concern in stage MS with liver involvement is mass effect causing respiratory compromise — Dan von Allman
17:58Stage MS without distress can be treated with aggressive observation — Erika Neumann
17:58Once respiratory compromise begins, treatment options include chemotherapy, radiation, or emergent decompressive laparotomy — Erika Neumann
19:06Classic findings of stage MS (high catecholamines, blue blebs on skin, liver metastasis, adrenal mass) may not require biopsy — Dan von Allman
19:52Liver biopsy in newborns is difficult because bleeding is hard to control — Erika Neumann
20:40If NMYC is amplified in stage MS, staging changes from MS to M — Todd Ponsky
21:05VIP secretion can cause severe diarrhea in neuroblastoma — Erika Neumann
21:05Initial workup for abdominal mass includes ultrasound to determine solid vs cystic, then CT with PO and IV contrast if solid — Erika Neumann
22:56Ultrasound is important for Wilms tumor to assess venous extension — Tony Sandler
23:50Large mass encasing aorta and celiac axis with microcalcifications represents L2 INRG classification — Tony Sandler
23:50Complete staging workup includes bone marrow biopsy, MIBG scan, chest CT to rule out metastasis, and head CT if clinical symptoms present — Tony Sandler
25:0610% of neuroblastomas are not MIBG avid — Dan von Allman
25:06PET scan may detect metastases in MIBG-negative neuroblastomas — Dan von Allman
26:03PET scan is not part of routine initial diagnostic workup but may be used for MIBG-negative soft tissue areas to distinguish recurrence from scar — Erika Neumann
27:10Open retroperitoneal biopsy provides adequate tissue size for pathology and biology studies — Tony Sandler
27:10Transperitoneal laparoscopic biopsy may not allow adequate bleeding control for large tumors — Tony Sandler
27:10Multiple percutaneous biopsies may not provide adequate tissue for biology studies — Tony Sandler
27:10NMYC amplification can be obtained from bone marrow, but additional biology studies require tumor tissue — Tony Sandler
28:28Biology studies beyond NMYC include ALK mutation and ploidy status — Tony Sandler

Cases discussed

1:2926-week fetus with prenatal diagnosis of left-sided suprarenal mass, managed with observation protocol
12:27Child with observed prenatal adrenal mass that resolved, later presented with metastatic disease
13:38Observed prenatal mass that grew at 12 months, resected and found to be pulmonary sequestration
17:00Newborn with stage MS neuroblastoma (skin lesions and liver metastases)
21:05Three-year-old with large central abdominal mass discovered during bath

Points of disagreement

19:06Necessity of biopsy in classic stage MS presentation
- Dan von Allman: With classic findings (high catecholamines, blue blebs, liver mets, adrenal mass), biopsy may not be necessary
- Erika Neumann: Biopsy may be needed to determine if NMYC is amplified, which changes staging from MS to M
27:10Approach to tissue acquisition for large abdominal mass
- Tony Sandler: Prefers open retroperitoneal biopsy for adequate tissue and bleeding control; reticent about laparoscopic transperitoneal approach; percutaneous may not provide adequate tissue for biology
- Todd Ponsky: Implied preference for avoiding transperitoneal approach in neonates to decrease risk of abdominal compartment syndrome

Open questions

What is the optimal size threshold for surgical intervention in observed prenatal neuroblastoma (5 cm vs 6 cm mentioned)?
Should PET scans be routinely obtained for the 10% of neuroblastomas that are MIBG-negative?
What is the best biopsy approach for large abdominal neuroblastoma: open retroperitoneal, laparoscopic, or percutaneous?
How long should surveillance continue after resolution of a prenatal neuroblastoma given the case of delayed metastatic presentation at age 3?
Is bone marrow tissue adequate for all necessary biology studies or is tumor tissue always required?

This episode was analyzed and enriched by Kai, the Library's AI content creator. Every item links to the moment it comes from — click a timestamp to listen in context.

Written for:

Neuroblastoma Management: From Prenatal Mass to High-Risk Disease

The episode's teaching points arranged as a structured lesson, building from the basics up to the finer points. Written by Kai from the episode transcript and reviewed before publishing.

For trainees · Teaching arc · AI-written, human-reviewed

Prenatal suprarenal mass: observation over intervention

When ultrasound identifies a suprarenal mass before birth, adrenal hemorrhage is more common than neuroblastoma — especially when fetal stress is documented 2:11. Other possibilities include pulmonary sequestration below the diaphragm and misdiagnosed renal anomalies 2:11. Radiologists can often distinguish adrenal hemorrhage on ultrasound alone 6:02, but the first postnatal study should still be ultrasound of the abdomen 5:07. If the mass is small (around 3 cm) and urine catecholamines are normal, cross-sectional imaging is not needed 5:34. Elevated catecholamines prompt an MIBG scan 6:02.

The Nocktern study established that prenatal neuroblastoma can be safely observed with intensive surveillance 8:44. Of 84 patients managed this way, only 16 (approximately 20%) required resection for growth or family preference 10:39, yet event-free survival was 98% and overall survival was 100% 11:07. The surveillance protocol is dense in the first year: ultrasound and catecholamines at birth, 3 weeks, 6 weeks, 12 weeks, then spaced out to one year 11:37. After the first year, intervals extend to every six months, then yearly 11:37. Surgery is indicated when the mass reaches 5 cm 14:36, volume increases by more than 50% 15:08, or catecholamines (VMA or HVA) rise by more than 50% 15:08. Even when growth prompts resection, the tumor is still likely to be low-risk [q3]. One caution: a case exists of a child whose observed prenatal mass resolved, who then presented at age three with widely metastatic high-risk disease 12:27.

Stage MS: when metastatic disease is not high-risk

Stage MS neuroblastoma (formerly 4S) — newborn disease with liver, skin, and bone marrow metastases 8:14 — still tends to have favorable biology 17:30. The primary threat is not the metastases themselves but mass effect from hepatomegaly causing respiratory compromise 17:58. If the infant shows no distress, aggressive observation is appropriate 17:58. Once respiratory compromise begins, options include chemotherapy, radiation, or emergent decompressive laparotomy 17:58. The classic presentation — elevated catecholamines, blue skin blebs, liver metastasis, and adrenal mass — may not require biopsy 19:06, though liver biopsy in newborns is difficult because bleeding is hard to control 19:52. One critical exception: if NMYC is amplified, staging changes from MS to M, converting a favorable-biology scenario into high-risk disease 20:40.

High-risk disease: biology over anatomy

For a three-year-old with a large central abdominal mass, initial workup starts with ultrasound to determine whether the mass is solid or cystic 21:05. If solid, CT with oral and IV contrast follows. A large mass encasing the aorta and celiac axis with microcalcifications represents L2 classification in the International Neuroblastoma Risk Group system 23:50. Complete staging includes bone marrow biopsy, MIBG scan, chest CT to rule out metastasis, and head CT if clinical symptoms are present 23:50. Ten percent of neuroblastomas are not MIBG avid 25:06; PET scan may detect metastases in these cases 25:06, though it is not part of routine initial workup 26:03.

Lymph node status in neuroblastoma does not drive treatment decisions the way it does in Wilms tumor 16:36. Biology is what matters 17:00. Open retroperitoneal biopsy provides adequate tissue for both pathology and biology studies 27:10, whereas transperitoneal laparoscopic biopsy may not allow adequate bleeding control for large tumors 27:10, and multiple percutaneous biopsies may not yield enough tissue 27:10. NMYC amplification can be obtained from bone marrow, but additional biology studies — including ALK mutation and ploidy status 28:28 — require tumor tissue 27:10.

The shift from anatomic staging to risk stratification based on tumor biology is the conceptual pivot that separates modern neuroblastoma management from older paradigms. A newborn with liver metastases may need only observation, while a three-year-old with a localized tumor may require multimodal therapy — the biology, not the extent of disease, determines the path.

Takeaways from this story

Prenatal neuroblastoma can be safely observed with 98% event-free survival; surgery only for 5 cm size or >50% growth.
Stage MS with liver mets has good biology; primary risk is respiratory compromise from hepatomegaly, not metastases.
NMYC amplification in stage MS changes staging to M, converting favorable biology to high-risk disease.
Lymph node status does not drive neuroblastoma treatment; tumor biology (NMYC, ALK, ploidy) determines therapy.
Open retroperitoneal biopsy provides adequate tissue for biology studies; percutaneous may not yield enough.

Tissue Acquisition in Neuroblastoma: When Biology Demands More Than Diagnosis

The points where the speakers disagreed, with each position presented side by side. Written by Kai from the episode transcript and reviewed before publishing.

For specialists · Points of disagreement · AI-written, human-reviewed

The Question

A three-year-old presents with a large central abdominal mass encasing the aorta and celiac axis — L2 by INRG classification 23:50. Cross-sectional imaging is complete, MIBG is positive, urine catecholamines are elevated. The diagnosis is neuroblastoma. The question is not whether to obtain tissue, but how much tissue is required, and by what route.

The Case for Open Retroperitoneal Biopsy

Tony Sandler's position rests on tissue adequacy for the full panel of biology studies that now drive risk stratification and therapy selection. An open retroperitoneal approach provides a specimen large enough for pathology, NMYC amplification, ALK mutation analysis, and ploidy determination 28:28. Multiple percutaneous passes may not yield sufficient tissue for this panel 27:10. Bone marrow can provide NMYC status, but the additional biology studies require tumor tissue 28:28. The retroperitoneal approach offers direct visualization and control of bleeding from a highly vascular tumor 27:10. A laparoscopic transperitoneal biopsy in a neonate or young child risks inadequate hemostasis and, in the setting of liver involvement, potential abdominal compartment syndrome postoperatively 27:10.

The Counterposition: Minimizing Intervention When Diagnosis Is Clear

Dan von Allmen and Erika Neumann articulate a more selective approach. In stage MS disease with classic findings — elevated catecholamines, blue cutaneous nodules, liver metastases, and an adrenal primary — biopsy may not be necessary at all 19:06. The clinical picture is diagnostic. The primary concern in these infants is not histologic confirmation but respiratory compromise from hepatic mass effect 17:58. If intervention becomes necessary, the easiest accessible lesion should be sampled, but the liver itself should be avoided because neonatal hepatic bleeding is difficult to control 19:52. The implicit argument is that when the diagnosis is secure and the biology is likely favorable, the morbidity of open biopsy is not justified.

Neumann raises the one scenario that changes this calculus: if NMYC is amplified in what appears to be stage MS, the staging shifts from MS to M, and treatment intensity escalates 20:40. This is the biological threshold that matters. But NMYC can be obtained from bone marrow 28:28. The question becomes whether the additional biology studies — ALK, ploidy — alter management enough to justify the morbidity of open biopsy in a neonate with classic MS presentation.

Where They Agree

Both positions acknowledge that biology drives therapy more than anatomic stage in neuroblastoma 17:00. Lymph node status does not change treatment the way it does in Wilms tumor 16:36. The tumor's molecular characteristics — NMYC amplification, ALK mutation, ploidy — determine risk stratification and chemotherapy intensity 28:28. Both agree that if a decompressive laparotomy is required for respiratory compromise in stage MS, tissue should be obtained at that operation 17:58. Both agree that percutaneous biopsy has limitations in tissue yield 27:10. The disagreement is not about whether biology matters, but about how much tissue is required to obtain it, and whether the clinical scenario permits a less invasive approach.

What Would Resolve It

The discussants do not explicitly state what evidence would settle this question, but the resolution lies in data on tissue adequacy. If bone marrow plus a single core biopsy reliably provides sufficient material for the full biology panel — NMYC, ALK, ploidy — then the case for open biopsy weakens. If registry data show that inadequate initial tissue leads to repeat procedures or delays in risk stratification, the case for open biopsy strengthens. The other variable is institutional: if the pathology and molecular laboratory can extract full biology from limited tissue, the threshold for open biopsy rises. If they cannot, it falls. This is a question of tissue requirements meeting surgical morbidity, and the answer may differ by center.

Takeaways from this story

Open retroperitoneal biopsy provides adequate tissue for NMYC, ALK, and ploidy studies; percutaneous may not.
In stage MS with classic findings, biopsy may be unnecessary unless NMYC status changes staging from MS to M.
Neonatal liver biopsy risks uncontrolled bleeding; if tissue is needed in MS, sample the easiest accessible lesion.
Biology (NMYC, ALK, ploidy) drives neuroblastoma therapy more than lymph node status or anatomic stage alone.

Keywords

neuroblastoma adrenal hemorrhage pulmonary sequestration Wilms tumor stage MS neuroblastoma stage 4S neuroblastoma high-risk neuroblastoma metastatic neuroblastoma respiratory compromise abdominal compartment syndrome ultrasound CT scan MRI MIBG scan PET scan

Hashtags

#neuroblastoma #pediatric surgery #pediatric oncology #prenatal diagnosis #super renal mass #childhood cancer #cancer #Cincinnati Childrens Hospital #PediatricOncology #PediatricSurgery #PrenatalDiagnosis

Transcript

Welcome to the latest episode of Stay Current in Pediatric Surgery, a multimedia production designed to spread the latest knowledge freely. This chapter is created and edited by Todd Ponsky, Alex Kessar, Alex Gibbons, and myself, Ray Hanky. Does your head spin when you think about neuroblastoma, low, intermediate, high risk and how the management changes for each? Join us as we break down this complex subject with pediatric oncology experts from around the country. Welcome to Stay Current in Pediatric Surgery. This is Todd Ponsky recording live from Cincinnati Children's Hospital. And today we're going to be talking about a topic that I don't know much about and we get to hear the real experts tell me about what's new in neuroblastoma. Neuroblastoma is a common problem that we all deal with and it seems like the the way to manage this is changing day by day. So we finally got some experts to put some clarity on this. First, I'd like to introduce Dr. Dan von Allman, who is the surgeon in chief here at Cincinnati Children's Hospital and is our expert when it comes to neuroblastoma. We also have Dr. Erika Neumann, who is at CS Mott Children's Hospital in Michigan and she has a clinical focus on oncology. And finally, we have Dr. Tony Sandler, who is the surgeon in chief at Children's National Medical Center and he also has a focus in oncology and neuroblastoma. Let's dive right in. We're going to, just like we always do, we'll pick some cases that are pretty common and sort of hit on some of the the nuances of the disease. Dan, you're in clinic and you are seeing a prenatal consultation with a woman that has a 26-week fetus that has a prenatal diagnosis of a left-sided super renal mass. Otherwise, the ultrasound is normal. What do you do with that? What is your differential diagnosis with a left-sided super renal mass? Uh, the most common things we think about, first would be an adrenal hemorrhage. That's more common if there's some history of stress to the fetus or something like that, but certainly that's one of the more common things that we think about. And then obviously, if it's solid or cystic mass, you also have to consider the possibility of a neuroblastoma. The other differential diagnosis include things like uh pulmonary sequestration, which can occur below the diaphragm as well as potentially a renal anomaly that's been misdiagnosed as a super renal mass. So, Erica, what do you tell the family now? So they come to you, you've got this super renal mass, you don't know what it is. How do you counsel them? So, I mostly I'm quite reassuring at this stage. It's still a bit early. I think you said the patient was at 26-week gestation? Yes. Yeah, so it's a little early. We still have some time to go before delivery. And I don't really do anything before the delivery. I don't get any more studies and when the baby is born, then I'll ask the family that if it's not born at our hospital, I'll ask the family to please get in touch with me. If it's here, I'll say, let me know when the baby is born and I'll come up and meet the family and the and the baby after the baby is born. I think it's important to reassure and help the mom understand that the priority during the pregnancy is the mom. And once the baby is born, then we'll learn more and the first thing we'll do is get more information once the baby is born. Tony, would you add anything else to that in your consultation? Yeah, I agree very wholeheartedly with Erica and reassurance. I think often when we're doing these prenatal diagnoses, we get too carried away with the details of the disease and uh we get parents all frightened about it. The other thing that I think is important to remember is to ask the family whether there's any familial history of neuroblastoma. I know it's very rare and it only occurs in about 1% of patients, but a familial history of neuroblastoma would help probably guide the diagnosis of a super renal mass in this mother. That's a great point. Okay, so you did that, Tony. Now what? Well, the the obviously the the pregnancy should go along as expected. The mother should uh have a normal prenatal care. And then once the child is born, we start uh doing some postnatal studies. And of course the first one that you would do is examine the child making sure that this is a otherwise healthy child that's asymptomatic. And if that is the case, then I'd probably start off with an ultrasound of the child's abdomen. The child then is born at 38 weeks. You would get the ultrasound. Um, would you get any CT scan other MRI or any other axial imaging, Tony? Uh, I wouldn't unless um the urine caticolamines are are high and unless you're concerned about something unusual. If it's depending on the size of the lesion, if it's probably let's, let's for argument's sake, call it a 3 cm lesion. I would probably just follow with ultrasounds and urine caticolamines. If the caticolamines are high, then one, we can start discussing whether perhaps an MIBG would be worthwhile, and that would be my next step if I was going to work this up further. I would agree completely with Tony. I think that the, you know, the ultrasonographers and the radiologists are actually quite good at at identifying uh what looks to them like a adrenal hemorrhage. And you can, while you can't be sure, uh it certainly helps to get the urine caticolamines to uh direct the counseling of the parents one way or the other. If the urine caticolamines are high, then you're going to want to counsel the parents about potentially more likely neuroblastoma. But if they're normal and the ultrasonographer is convinced that this is an adrenal hemorrhage, you can really be very reassuring, which doesn't mean you shouldn't follow it, but you can be um, you can be more definitive with the family about the risk of a tumor. All right, so I'm going to repeat what I hear so far up to this point. Reassurance during the fetal period. All you need to get is the ultrasound. After they're born, you get a follow-up ultrasound postnatally and urine caticolamines. Maybe if those are elevated, you would get an MIBG, but no need for a cat scan at this point. Any need for a bone marrow biopsy? You know, the only time I would do a bone marrow biopsy is if the MIBG is positive showing bone metastasis. Then I think it's worthwhile doing a biopsy because at that point you may want to determine whether this is a Nmic amplified neuroblastoma, which in all reality in these prenatal diagnosis is extremely, extremely rare. At this point, you have an ultrasound and urine caticolamines that are elevated. Tell me about the MIBG scan. What are you looking for on the MIBG scan, Erica? I'm mostly looking to characterize the primary lesion and then also to understand if there're any other areas of disease. What are the more common areas for metastasis? So, in a neonate or in the perinatal phase, the most common areas would be liver, bone, skin, and lymph nodes. All right, so, Dan, you have an MIBG that lights up only at the super renal mass. Yep. Uh, do you observe or do you operate? So, uh that is a a discussion you have to have with the family uh because the uh data from the Nocktern study would suggest that you can confidently observe these patients uh, but you have to observe them carefully with ultrasound studies looking for growth, but that many of these patients will actually be spared any surgery if you just watch uh even if you have a by laboratory studies that confirm neuroblastoma with an MIBG and positive urine caticolamines. On the other hand, families are sometimes unwilling or uncomfortable uh having a mass in their newborn and they want it out. But I would suggest that probably most if counseled with the current data would be willing to simply observe this. Yeah, I agree with that completely. I think that Dr. Nocktern's studies have been very reassuring that um particularly in neonates with proper surveillance that most of the children don't require surgery or chemotherapy. For the first year of life there's a lot of imaging and monitoring that goes on, but um most of these children can be spared surgery. I think one of the other things when you speak to the family, it's really important to reassure them that even if the mass increases in size and you take it out, it is likely to be a very, very low risk tumor. So it's very uncommon to have a bad neuroblastoma. It's possible for sure. Yeah, appears in a newborn. A new born. As you're watching because cereal ultrasound studies and it begins to increase in size and you'll like to take it out, it's still likely to be a low risk too. Okay. All right. Todd, if I can also take a shot at that, the the number that I quote the families from that original study is that they um, of the, I think it was 84 patients that they observed, 16 underwent resection for either growth or some uh other reason, in other words the families didn't want to wait. And so that's about a 20% resection rate of those of ones that underwent observation. And of those patients, um, I think it was like about 98% had event free survival and 100% had overall survival. So you're quoting a study that was done uh with very good outcomes and I think it's very reassuring for the families to hear that there's data. Great. Agree. Yeah. So how often do we check ultrasound and urine caticolamines and for how long? So it's pretty intense for the first year. Um, they're getting them at birth, then at three weeks, six weeks, 12 weeks and then as long as things are stable, then they get spaced out from there all the way to one year. Then we stop at a year. Then a year it becomes something more like every six months. After every six months, then a year, I think it's more than up to the oncologist how they would ordinarily do that. But I do think that if they're followed by the oncologist, they tend to be followed long-term, not long-term, but they they're followed for probably more than a year. Um, many of the lesions resolve. So they're gone and once they're gone, then you get some ultrasounds after that to make sure they're gone. The only caution I would raise is that we had a case of a child who presented with exactly this scenario, was observed, the adrenal mass went away and age at age three she presented with widely metastatic high risk neuroblastoma. Wow. So that's a cautionary tale, but I I still think that the overall outcomes are really, really good and you should just observe these. Dan, that is a very interesting case. Um, you know, I that does actually bring caution to our general approach to managing these. The other thing that I'm always um a little bit cautious about, I'll say is when I'm counseling the patients and as we're getting through this first year, it's always a little bit um unclear to the families because we don't have a biopsy and really what we're going by is how the lesion looks on the ultrasound and on the CT scan and the caticolamines, but you don't know really what the tissue diagnosis is until we in when and if we take the mass out. And so I'm always cautious in saying, like all the data we have, it looks like a neuroblastoma. But I will say the last one of these that I had at the 12-month scan it got bigger. And we took it out and it was a ended up being a sequestration. And so I was glad that we had kind of prepped the family that this is most likely neuroblastoma but it could be something else um because after going a year thinking your baby has a neuroblastoma and then you end up with pulmonary sequestration that was below the diaphragm, you know, we just I I think it's just important to make sure that we are very reassuring to the family that based on all the information we have, this is what this probably is, but that we have to have some level of uncertainty because we we don't biopsy these kids. So, the only thing that I would discuss is when does one operate and I'm not exactly sure what the size is. I've always used the 5 centimeter size as an indicator for surgery, but I'm not sure um, that is sort of my cutoff. If it's obviously getting smaller, I wouldn't operate. If it's getting bigger, I do operate, but I'm not sure what my other colleagues on this esteemed panel, what size they use. I I would agree with you uh Tony. I think that if the mass, first of all, if it's increasing in size, then that in and of itself is concerning and there are specific criteria, but once a mass gets to be 5 centimeters, then I I personally would be recommending to the family that we take it out. I agree with that and then as we're surveying, I use the original criteria of, you know, if it goes increases in more than 50% volume. And then the other thing we look at are the urine caticolamines. And uh 50% increase in either the VMA or HVA would prompt us to start thinking more about surgery. That's a great point, Erica. That's I think it's important that you're not just doing ultrasounds, you're continuing to monitor urine caticolamines. So, uh Dan and I just came up with the same exact question at the same exact time. So Erica, if you do decide to remove it, would you do it laparoscopic or open? Yeah, I think laparoscopic, if particularly if it's less than 6 centimeters, I think it's worth a shot to put a scope in and take a look. Um, I was glad we were able to do that for this baby. I think that sequestration ended up being about 3 and a half centimeters. And it came out very nicely with a laparoscope. The child went home the next day. Do you need to do any lymph node sampling? I don't think lymph nodes give us any more information than we already have in neuroblastoma from the cross sectional imaging. I agree completely. Uh unlike Wilms tumor, the lymph node status of neuroblastoma, um is not as important in the change of therapy. The biology of the neuroblastoma, which I'm sure we'll discuss later is more important than lymph node status. Erica, let's say the child has skin lesions and liver mets. Now what? Now things get a little different. This would what we're calling now staging the old stage 4S and I would say I'm still reassuring at this point with the family. The biology of these tumors tend to still be good. These aren't bad neuroblastomas. There is a scenario where things could get bad and that's if the liver is filled um and engorged with tumor. What what exactly can go wrong when the child has a liver full of tumor? The most common thing that we worry about is is the mass, is the mass effect of the tumor in the liver, which can be really dramatic and causes respiratory compromise. And that's the problem that the a newborn would face is increasing uh ventilatory requirements and uh inability to ventilate once the tumor gets large enough. So how do we treat these these children? If they're not in any distress, they're treated with my favorite protocol, which is aggressive observation. That is you don't necessarily have to uh you don't necessarily have to do anything other than watch them, but once they begin to get in trouble, then you certainly want to have tissue and make sure that it is in fact MS and not a high risk tumor and you can treat them with either chemotherapy or radiation if you get to it early enough. If the child acutely decompensates, then doing a decompressive laparotomy is also described as a potential emergent therapy for kids who have respiratory compromise. See, you mentioned if we have tissue. So it is not a requirement to biopsy even the skin lesions in these children? I think that in most cases and I'd be interested to hear what what Erica and Tony say, we want, we would get tissue to confirm the diagnosis and get some biologic information. Yeah, I think uh if you have the classic findings with uh high urine uh epinephrin, norepinephrin or caticolamines. And you have the blue blebs on the skin uh with a liver with liver metastasis and a uh adrenal mass, I don't think you really even have to biopsy that kid. However, if you're taking the kid to do something radical like a laparotomy uh to place a silo to decompress the abdomen, then I would certainly biopsy the easiest lesion. I'm not sure I would biopsy the liver uh because once you get bleeding in a in a newborn liver, it's very hard to control. So I would take another lesion other than the liver unless the liver is the easiest to get to. But yeah, I completely agree with Dan, it's uh observation. I haven't in my career had to open an abdomen and place a liver in a bag or anything like that. I agree. Um, I know that our oncologist have often asked us to do a biopsy and they like to get that because if in the rare instance the Mican is amplified, it changes them then from MS to M. And so for that we're sometimes faced with should we be going into the abdomen? If we can avoid particularly in neonates going into the abdomen for a biopsy, I think we can decrease the risk of that abdominal compartment syndrome postoperatively. So, Erica, Tony, let's talk about another case or another child that we that we often times see and this is a three-year-old who presents with a large central abdominal mass. You know, the classic history, grandma noticed in the tub giving the child a bath and they come in uh with this large abdominal mass, otherwise healthy child. How would you work that kid up? Erica, what's your approach? First, obviously, we do a good history and physical exam, finding out if the child has had any other illnesses, any family history, past medical history. Uh, I saw a child today that had diarrhea. I thought that was interesting because we, remember I've always read that you can get, you know, VIP secretion and severe diarrhea from neuroblastoma, but I had not seen that and I always ask about that. And then with respect to trying to to understand more about the mass, I'll typically start with an ultrasound. Um, I get an ultrasound to to see if I can get a clue on whether this is a solid or a cystic mass. If it's solid, then most likely we would then move on to cross sectional imaging. Um, and I usually get a CT scan with PO and IV contrast just because I'm more comfortable reading a CT scan. While we're waiting on the CT scan and and the contrast, we'll get some blood work. Uh routine blood work is, you know, CBC, a comprehensive panel. Um, I'll usually get um tack on uh amylase lipase LDH and just a standard blood work that we would be getting for anyone that would have some um abdominal masses. And so, um, we then send the urine caticolamines and I'd wait and see what what all that showed. Great. Um, Tony, anything you would add to that initial evaluation? No, I agree completely. Um, you know, when you're initially seeing a child with an abdominal mass and you're not sure what it is, I think it's very reasonable to go ahead and get an ultrasound. Uh if it turns out to be Wilms tumor, the ultrasound is already done because you want to see if there's any venous extension of the tumor. And as Erica clearly stated that it you also want to be able to tell whether this is a cystic or solid mass. Yeah, that venous extension thing for the Wilms is a great point. I that's uh that's a great reason to get the ultrasound. But so you get a CT scan and it shows a big 10 cm mass that encases the aorta and the celiac axis. Micro calcifications, more solid than cystic. Now what would you do? Uh Tony, what's your group's workup? So Dan, what you're describing uh is an L2 international neuroblastoma risk group uh classification of the local tumor. So this is obviously going to need necessitate a complete workup and obviously the oncologist now get involved and are really key to the the further workup, which includes uh uh probably a bone marrow, um the the CT scan already being done. You're getting a technetium scan or MIBG scan. Um, I also like a CT scan because I can read it better. An MRI is certainly an option. You also want a chest CT because you want to rule out metastasis. Uh and then also a head CT can be indicated if there's any clinical symptoms that the patient may have. So now you're talking about the staging of the disease and then of course the final aspect to staging the disease is getting a biopsy uh for pathology and and of course the biology in the case of neuroblastoma. So how about pet scans? Do you do either of you guys get pet scans? Our guys are very fond of pet scans. We're also very fond of pet scans, but the question is for the initial workup, a pet scan is not going to be my workup. An MIBG scan will be. And I'm not sure a pet scan will have any benefit over an MIBG scan in neuroblastoma. Correct me if I'm wrong. No, I think, I think you're right. I agree with you. I think the rationale that's often given is that 10% of neuroblastomas are not MIBG avid. So potentially they would be pet avid even though they're not MIBG avid and you might also pick up uh metastasis that way. So I agree with you. It's sort of uh we get them and we get lots of them, but um, but it may or may not be necessary, especially if it's MIBG avid then you have a study that you need to not only make the diagnosis and identify mets but also to follow it, I think. Dan, I think you outlined that clearly. If it's certainly if it's 10% and your MIBG is negative, then I would agree with pets, I wouldn't argue against a pet scan. Erica, how about you? Do you guys get them? No, we don't get them. They're very few scenarios we get them where um every now and then we've we have a child where the primary lesion has been resected and there's a funny looking um soft tissue area that is MIBG negative and we're trying to decide if this is a local recurrence or um nothing or scar. And so they'll get a pet scan in a scenario like that, but that wouldn't be part of our routine initial diagnosis workup. Uh Tony, you mentioned getting tissue, obviously it's the sort of the key or final step in doing the staging. What do you think about the options for for getting tissue? Do you and and Erica, do you do y'all do percutaneous biopsies? Do you do laparoscopic biopsies? Do you do open biopsies? Lots of different ways to get tissue. So, uh Erica, I'm sure you'll probably contradict me, but this is sort of my approach. Um, I'm sort of old fashioned in this way because I've been, you know, trained to get that size of tumor that can be biopsied uh safely and that can uh give the necessary tissue uh for further studies and bio biology of the tumor. And so I've always done a retroperitoneal approach to a large mass and done an open biopsy. I am uh a little reticent to do a transperitoneal approach and biopsy a big tumor uh laparoscopically because I'm not sure I can control the bleeding as well as a retroperitoneal approach. You could do a percutaneous biopsy. You would have to do multiple uh uh percutaneous biopsies. Um, and I'm not sure um the tissue collected is adequate for biology. So, uh people also say you can take bone marrow and you can get the Nmic amplification on bone marrow, but there's more to do than just Nmic. There's the alktion to look for. There um is of course adequate biology for pathology. And of course you um, you know, you really do want uh to look at the deployed status of the tumor as well. So, right now I am still doing open biopsies unless there's some reason that the child is very sick and they can get Nmic amplification of the bone marrow. That's been my strategy right now. I'm sorry for the long-winded answer. No, that's awesome. That's great. Can you explain to me what you mean when you say you do a retroperitoneal biopsy?

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