Please send as an e-mail (stokman1@cox.net) letting us know if you contributed. We would like to acknowledge everyone's contribution and confirm that it gets to the correct place.Our long-term goal is to create a research fund that will focus exclusively on JXG. This is a major undertaking, which will require a great deal of time and effort, but could have far-reaching impact in the future. If you would like to support this effort with a contribution, please make your cheque payable to Histiocytosis Association of America and be sure to write following note in the memo area: "In memory of Sydney Salem Golding, designated for the research fund." You can mail your contribution to: Histiocytosis Association of America, 302 North Broadway, Pitman, NJ 08071
Q. Does Ben have cancer? (Is JXG or LCH cancer?)
A. We do not clearly know the answer to that. Technically no, but for all practical purposes, yes. LCH & JXG are currently not considered cancers. But whether these histiocytic disorders are 'cancerous' or not is still hotly debated. Researchers are still not sure if one cell is going crazy (like cancer) or if the body is telling the immune system to deploy these specialized cells. Some recent evidence suggests that the disease may be cancerous, they have found that the cells in the lesions are the same cell or clonal, meaning one cell is reproducing incorrectly - this is like cancer.
To understand what that means to Ben, we need to understand two terms, Neoplastic and Reactive. If LCH or JXG were to be classified as neoplastic, we would think of them as cancer. If LCH or JXG were to be classified as reactive, we would not call them 'cancer'. Currently most histiocytosis are thought to be reactive (but remember, this is debated), so what is the difference?
Neoplastic is nearly synonymous with cancer (as far as I can tell). The word roots for neoplasm means new (neo) formation (plasm) and it has come to mean abnormal growth. When something is neoplastic or cancer, it means that cells are reproducing, usually at a very fast rate. The cells are not behaving like they are suppose to. Somehow, at the gene level, something has gone wrong and the instructions for normal cellular activity have gone awry.
Reactive processes are slightly different. In my lay understanding, instead of cells misbehaving, it is the immune system that is misbehaving which in turn causes cells to misbehave. The immune system is sending its histiocytic cells - the cells then go proliferate at a high rate and look and act like what you and I consider cancer. The immune system is sending signals to tell the cells to do their job in the wrong place at the wrong time and at the wrong 'volume'.
What does this mean to Ben? From Ben's perspective (and ours) there is almost no difference from what we typically consider cancer. We see cells producing in the wrong locations at a much higher than normal rate. These cells then start to impact normal operation of the body. So in the final analysis, it looks like cancer, acts like cancer, is treated by cancer specialist (oncologists) who use the same medicines they use for cancer (chemotherapy). Technically we can not call it cancer, and in some cases it may be worse than cancer because it is not as well understood as many types of cancer.Q. Are Ben's lesions malignant or benign? (Are JXG or LCH lesions malignant?)
A. We know that Ben has abnormal bodies growing in his body in several locations (brain or central nervous system, lung, scalp, & eye bone - called the orbit), but how are these bodies acting and what are they doing to Ben? We do not know the answer with clarity. We often get the question are the lesions benign or malignant? We need to understand these terms slightly better to understand the impact to Ben. When something is benign, we typically think of it as self-contained. When something is malignant, we typically think of it as going outside itself, typically through the bodies blood processes. I like to think in terms of the micro and the macro levels - at a micro level, a tumor may be considered benign - it does not seem to be spreading outside itself, but at a macro level, there could be several of these tumors. This no longer sounds benign. Also, you can have tumors that appear benign, but they can be located in very critical places and their impact is no longer 'benign'. For Ben's brain tumors or lesions, it is very difficult to classify them as benign or malignant for the following reasons:
There are multiple tumors / lesions (sounds malignant)
They appear to stay within their own 'boundaries' with nice rounded shapes (sounds benign)
They are secondary - there is no single tumor and this implies some source (sounds malignant)
They are in potentially life threatening places (sounds malignant)
So you might be able to classify them as either benign or malignant, but that does not give us any real information - the important factor is that they are in very high impact locations, they can effects Ben's quality of life and they are growing.Note: when people say cancer, they are generally referring to malignant neoplastic prolifrative processes.
Q. Does Ben have tumors or lesions in his brain? (Are tumors and lesions different?)
A. Should we call what Ben has in his head tumors or lesions? It does not really matter - they look and act like tumors. Technically from the reactive vs. neoplastic discussion above, we should probably call them lesions. So what are lesions? Basically anything they cannot really identify.
Q. What is a Histiocyte?
A. Word roots: histio- connective tissue, cyte - cell (so histiocyte loosely translates to 'tissue cell'). Tissue cell is not the true or correct definition. Histiocyte has come to mean white blood cells that have moved out of the blood into the tissue. More specifically a histiocyte is a resident, 'typically' non-moving, macrophage and/or perhaps a dendritic cell. Histiocytes perform many roles. When working normally a macrophage's job is to find material of a foreign gnome and then tell other immune system components to destroy the foreign material. Macrophages play an important role in killing of some bacteria, protozoa and tumor cells. Other histiocytes called antigen presenting cells or dendritic cells release substances that stimulate other cells of the immune system and are involved in antigen presentation. An antigen is a substance that stops foreign cells (or any antibody) from producing, Antigen literally means against (anti-) coming to be (gen). So simply, histiocytes are a normal part of a correctly working immune system. They quite often help save our lives.Q. What is Histiocytosis?
A. Word roots: histio- tissue, especially connective tissue, cytosis -a condition in which there is more than the usual number of cells (so histiocytosis loosely translates to 'abnormal number of tissue cells'). A condition marked by the abnormal appearance of histiocytes (macrophages and/or dendritic cells) in the blood and other connective tissue. Basically histiocytes and the immune system are somehow doing the wrong things.
To add to this, the term histiocytosis may not be the best name for these disorders. There has been much research on dendritic cells. Dendritic cells and not macrophages may be at the heart of these diseases. For example, the langerhans cell (the cell involved in LCH), is actually be a dendritic cell, not a macrophage. Macrophages and dendritic cells are closely related, but not the same. The name histiocytosis came into being because the langerhans cell is a tissue cell and many of the histiocytoses show tissue symptoms. A better name might have something with 'dendritic cell' in the title.
So how are macrophage-monocytes different from dendritic cells? As I understand it, they are both what are called antigen presenting cells (APCs). They pass off signals to other cells (T-cells - a specific type of lymphocyte) to let them know antibodies (foreign invaders) are in the body. They are mobile so they can travel in the body and then go to the lymph system to present the antigens and signal the T-cells to go attack. Macrophages (Macro-large, phage-eater, or large eaters) are also phagocytes (phag-eater, cyte-cell, or eater cells) - this means that they consume the antibodies (bacteria, fungi, viruses, foreign cells). Dendritic cells (named for their large and numerous arms that are able to reach between many cells to present antigens) are not really phagocytes, they are more specialized and focus just on antigen presentation. They do 'eat' foreign antibodies, but they really only do this to be able to present the antigen to the antibody to other cells. While macrophages travel to the site of infection, they tend to stay and eat the antibodies and foreign cells (true phagocytes), dendritic cells also travel to the site of infection, and eat the foreign invaders, but then they tend to travel back to the lymph system and notify other cells to attack.
All of this is the 'normal' operation. When these cells send the wrong signals or reproduce in incorrect numbers (at the wrong 'volume'), this is when we end up with histiocytic disorders. Remember, we do not know why this happens - yet.
Macrophage - a professional eater of foreign substances that also presents antigens to other cells to help out
Dendritic Cell - a professional presenter of antigens to other cells that also eats foreign substancesThe following was copied from the Histiocytosis Association of America web site:
Histiocytosis is a rare blood disease that is caused by an excess of white blood cells called histiocytes. The histiocytes cluster together and can attack the skin, bones, lung, liver, spleen, gums, ears, eyes, and/or the central nervous system. The disease can range from limited involvement that spontaneously regresses to progressive multiorgan involvement that can be chronic and debilitating. In some cases, the disease can be life-threatening.Q. What are the different types of histiocytoses?
In some ways, histiocytosis is similar to cancer and has historically been treated by oncologists with chemotherapy and radiation. Unlike cancer, histiocytosis sometimes goes into remission without treatment.
The vast majority of people diagnosed with histiocytosis are children under the age of 10, but it is also found in adults of all ages.
It is approximated that histiocytosis affects 1 in 200,000 children born each year in the United States. This illness is so rare, there is little research into its cause and treatment, and it is often referred to as an "orphan disease," meaning it strikes too few people to generate government- supported research.
Another histiocytic disorder is HLH (hemophagocytic lymphohistiocytosis), where there is an excess of both histiocytes and lymphocytes. This disease can be hereditary. Other histiocytic disorders include malignant histiocytosis and sinus histiocytosis.
A. There are several types of histiocytoses or histiocytic disorders. There have been several different ways to categorize them. The currently accepted system is as follows:
Class 1: LCH (Langerhans Cell Histiocytosis)
Class 2: Non-Langerhans Cell Histiocytosis
JXG (Juvenile Xanthogranuloma)
HLH (Hemophagocytic Lymphohistiocytosis)
Sinus Histiocytosis
Class 3: Malignant Histiocytosis
Leukemia
Histiocytic Lymphoma
Malignant Histiocytosis
Fibrous Histiocytoma
So LCH and JXG are both histiocytoses or histiocytic disorders. We do not know why and how they occur.Q. Are the histiocytoses a disorder of a specific type of cell?
A. You can group the non-malignant histiocytoses into dendritic cell disorders and macrophage cell disorders.
Dendritic Cell Disorders
LCH - Langerhans Cell Histiocytoses (Epidermal Dendritic Cell Histiocytosis)
JXG - Juvenile Xanthogranuloma (Dermal Dendritic Cell Histiocytosis)
Others...
Macrophage Cell Disorders
HLH - Hemophagocytic Lymphohistiocytosis
RDD - Rosa-Dorfman Disease (Sinus Histiocytosis)
Others...
We, Ben's parents, are focusing on Dendritic Cell DisordersQ. Is JXG a Histiocytosis?
A. Yes. It is different from LCH in that instead of involving Langerhans cells (probably abnormal Langerhans cells to be clear), it involves dermal dendrocytes (or dermal dendritic cells).Q. Why is JXG called JXG?
A. The disease is named after the skin lesions that often accompany it and after the fact that most of the people that get it are young. The skin lesions are usually a yellow to brownish-red color, with a raised waxy appearance. Here is the breakdown of the name:
J - Juvenile - Young
X - Xantho for Xanthoma or Xanthomas - The word “xanthoma” is made up of “xanth-“ from the Greek roots “xanthos” (yellow) and “oma” (swelling) = a yellow swelling. A xanthoma is a circumscribed yellow swelling, a yellowish nodule. A general term to describe fatty lipid filled areas under the skin. A benign lesion composed of lipid laden foam cells, which are histiocytes containing cytoplasmic lipid material. Also called xanthelasma and causes yellow lesions under the skin.
G - Granuloma - Chronic inflammatory lesion characterized by large numbers of cells of various types (macrophages, lymphocytes, fibroblasts, giant cells), some degrading and some repairing the tissues. A granuloma is a solid grouping of inflammatory cells coming together in a lump or solid structure. In its most classical form, a granuloma consists of concentric layers of cells that, together, form the distinctive lesion.Q. Is JXG the best name for the disease?
A. In my opinion, no. LCH used to be described by three different symptomatic presentations and led to a single disease being called by at least three different names (Eosinophilic granuloma, Hand-Schuller-Christian syndrome, Letterer-Siwe disease). This all changed when it was discovered that the Langerhans cell (the Epidermal Dendritic Cell) was central to all three diseases, the name was then changed from the old names to a name that indicated the underlying player - the histiocyte called the Langerhans Cell. The same could be done for JXG. The current name just describes how it looks in its skin presentation. The name could be changed to something that describes the underlying central player. This underlying player is suspected to be the Dermal Dendritic Cell. The disease could be called Dermal Dendrocytosis (for that matter, you could call LCH Epidermal Dendrocytosis).Q. How common are LCH and JXG?
A. They are both VERY rare and subsequently not very well understood. For LCH the statistics are quoted around 1 in 250,000 people will get it in some form or another. For multisystem JXG the occurrence rate is probably less than 1 in 10,000,000. What adds confusion to this is the different presentations of the disease. Many people get a single lesion, and in that form, the disease may not be life-threating. Ben has multiple system involvement (which is even more rare) - and one of those systems is the brain or central nervous system.Q. Does Ben have LCH or JXG?
A. Ben has JXG beyond a doubt. We know this from his pathology reports.Q. Are JXG and LCH closely related?
A. Yes, but how is unknown. There are several cases where a patient has both at one time, and cases where the disease changes from one to the other. Langerhans cells and dermal dendrocytes probably come from the same precursor cell. If something were to go wrong with the precursor cell, this might lead to having both of the diseases. Also, some chemical signals may be able to change langerhans cells into dermal dendrocytes or visa versa. These changes have been accomplished in the laboratory.Q. How do you diagnosis these diseases?
A. You can try to diagnose these diseases in two ways - from the symptoms & presentation or from the pathology.
Symptoms: Sometimes when backed into a corner, you can diagnose based on the symptoms. The common symptoms for LCH are scalp lesions, bone involvement (especially the long bones and the temporal & orbit bones in the skull). Common symptoms for JXG are not the orbit or bone involvement, but raised skin papula (unlike Ben's) and the eye itself (not the orbit). Ben seems to match LCH more than JXG.
Pathology: On the other hand, these diseases are usually diagnosed only by their pathology (what is going on at the cellular level). This is because there can be large variation in how the disease is presented. Pathology should be more reliable than just the clinical presentation of the disease.. Pathologist do stains and other analyses to see what is present. Ben's pathology is currently best aligned with something called 'early' JXG. We have not found Birbeck granules - which would prove LCH, and we have not found Tuton Giant cells which would prove JXG. For JXG, stains of Faschin and Factor XIIIa stain positive, Ben did not have these done, and we would like to have them checked. We have done a ton of reading on pathology, and I still do not understand all the details.Q. What is the differences between Multisystem and Single-system?
A. Diseases like LCH and JXG can effect multiple organ systems (skin, lungs, liver, spleen, lymph, bones & bone marrow, central nervous system....). Multisystem means more than one of these systems are impacted (usually not a good thing). You may also hear the term multi-focal which means there are lesions in more than one location in a given system. Multisystem & multi-focal is sometimes referred to as disseminated.Q. How do we treat Ben's condition?
A The whole spectrum of options available for cancer are considered. Radiation would have too many negative developmental impacts on Ben at this age and would therefore only be used as a last resort. Surgery could be successful for some presentations of this disease, but many of Ben's lesions in his brain or not accessible (dangerous locations). Chemotherapy is the most common approach. See the treatment thought process map.Q. What about surgery and radiation therapy for Ben?
A. Because the lesions are in Ben's brain, and because of their locations in the brain, surgery in not an option. We could access the main lesion to prevent hydrocephalus and thalamic invasion, but for the others, they are in locations too dangerous or with too great an impact to access. Radiation, especially for someone as young as Ben, is a poor option. It causes too much damage or sequelae. Ben's whole brain would be impacted.Q. Is JXG treated differently than LCH?
A. No, because of the lack of understand with JXG. There are only 36 reported cases of disseminated or multisystem JXG and therefore a very small experience base. Since is is somewhat similar to LCH, they treat it like LCH. This is true up to a point, you treat them the same early on. There are some drugs that may be better at tackling LCH than JXG and visa versa.Q. Do JXG and LCH go away after treatment with chemotherapy?
A. Hopefully, be we do not know. Responses are quite varied and do not clearly correlate with the presentation of the disease. Some people with several lesions get treatment and it never comes back. Others with few lesions get treatment and the disease does not respond and progresses to multi-system. The reasons are not known.
The first six weeks of treatment with chemotherapy are considered critical. We will be looking for a response in Ben in the first six weeks. If we do not get one, we will become more aggressive with treatment, and will use more powerful drugs. In general, a poor response after these first six weeks will be a bad sign.
One school of thought with those most familiar with JXG is that the lesions, especially CNS / brain lesions do not go away or respond to chemo. (This is not true for LCH, which have lesions that tend to respond to chemotherapy a little easier, but can come back a little easier or perhaps start new lesions.)Q. Does JXG respond to Chemotherapy?
We do not really know. We are hopeful. The following from a doctor with some JXG knowledge sums up the current knowledge:…What I have found … is that JXG is capable of presenting in just about any unique way possible. It is true that the clinical course for the vast majority of children with systemic JXG (that is, beyond the skin) is benign, meaning that the lesions will remain stable, perhaps shrink, but often simply remain the same size as the child grows "around" them. However, it is also true… that some children, especially infants, can have very serious medical problems up-front, requiring major supportive care while they outgrow their initial problems due to JXG.Q. Why might chemotherapy work?
In regard to treatment….(those) who have treated patients with JXG have been underwhelmed by the effectiveness of chemotherapy. Historically, surgical removal of the mass(es) has been the most reliable approach. But the problem is that for some infants, this just isn't feasible, due to the number and/or location of the masses. If their medical condition is stable, most people have found the best approach is supportive care, as indicated above. If their condition is not stable, most physicians would feel compelled to try treatment. One option is radiation therapy, but for infants this is not very appealing and, frankly, is of uncertain benefit, too. A more attractive theoretical option is chemotherapy. The problem here is that there haven't been any medications that have been clearly effective in shrinking these JXG masses. Most physicians…have utilized chemotherapy medicines known to be effective in Langerhans cell histiocytosis, a different but somewhat related disorder. … the responses to chemotherapy have been marginal at best, overall disappointing. Does treatment keep new lesions from growing? That is unknown; most observers note that patients with JXG seem to have all the lesions they will have when they are diagnosed, and do not typically grow new ones, although the existing ones can be difficult to treat in their own right.
Despite the overall good prognosis of JXG, there clearly are some patients who do die of complications of the disorder. From the medical literature, a large proportion of patients who die have lesions in their brains, which can be difficult to remove. …
A. I used to think chemotherapy worked by attacking fast growing or fast dividing cells. While this is true to some extent, I have been corrected, I now believe that chemotherapy is successful because the defective cells are somehow more sensitive. This increased sensitivity make them more susceptible to death in many ways (see how chemo works) This sensitivity also affords them some ability to change and this is why drug resistance happens. It is interesting to note that the same type of chemotherapies are used on cancers and many immune system disorders (e.g. rheumatoid arthritis). The Histiocytoses may be a sort of 'cancer of the immune system', therefore chemotherapy seems to be the right thing to do.Q. What things indicate success or a good prognosis?
A. Single system involvement (espically non-excutanious or only skin involvement), older age, good response in the first six weeks of chemotherapy. If organs are involved, it helps that they function well - organ dysfunction is a bad sign.Q. For Ben, what is the prognosis? What can we expect when the chemo works?
A. We do not know - and NO ONE has the ability to tell us. The complexity and lack of understanding of these diseases keep up from knowing. We have a six week window for chemo, at the end of that six weeks we hope and pray to see a reduction in the size of the lesions. Less favorable, but more likely would be a halt in the lesion growth. The worse case is that the lesions keep growing.Q. Why did we not start chemotherapy earlier?
A. There are several reasons for the five week delay from the time we found Ben's brain lesions to the time we started chemotherapy:
1) Much of the delay was due to the diagnostic process. We had to figure out if Ben had an infection (viral, bacterial or fungal) or if he had some sort of cancerous tumors. Several tests were performed to see what he had. All of the tests & biopsies for infection came back negative. He did not appear to have cancer either. He had something 'in between' - a malfunction of the immune system (why and how we do not know) that cause cancer like effects (lesions that act like tumors).
2) Once we knew we had a histiocytic disorder or disease, we needed to pinpoint which one. We did several more biopsies and the final results are still not back on this yet.
3) The type of biopsies we have taken are difficult to diagnose. Scalp lesions tend to 'burn out'. The brain lesions do not show presence of Langerhans cell - the pathology of the brain for LCH is very difficult.
4) We wanted to get Ben on the LCH III study if possible, so a definitive diagnosis of LCH would be necessary. In the end we could not wait, we needed to start chemo.
5) We wanted to confer with Dr. McClain and some of his counterparts to make sure that we would treat JXG the same way we would treat LCH. The conclusion is that we would (for lack of a better option or a good understand of JXG).
6) The pathology got sent slow mail from Dr. Dehner to Phoenix. This caused us to loose two weeks before Dr. Jaffe could review. In the end we did not wait on the final pathology report.Q. What are the long term impacts to Ben?
A. We are quite uncertain. Two things will impact Ben long term - the disease itself and the effects from treatment (chemotherapy, surgery or radiation).
From the disease itself, death is one we are, of course, vary concerned about. He could end up with learning disabilities - for some reason, people with LCH have some tendency toward depression and some learning disabilities. He could end up with some physical impact. LCH has a very high association with diabetes insipidis - LCH somehow attacks the pituitary which controls how much you drink and urinate. The other part of the pituitary controls growth - which would impact his height and size (see additional information below). Bone involvement can damage key bones. Lung involvement may impact his pulmonary abilities long term. We are not sure how many systems will be involved in the long term and what the impact of that involvement will be.
Chemotherapy will impact him as well, at his age it will impact his growth and overall height. He is obviously growing very fast at his age, and will be susceptible to the impacts of chemo. It could impact his mental abilities as well, but exactly how is unknown.
Or he could be just fine.Q. Can LCH and JXG come back?
A. Yes. After treatment and remission, LCH quite commonly returns. We would most likely treat with chemo again. JXG on the other hand, tends not to return with new lesions.Q. What causes LCH or JXG?
A. No one really knows. There are very tenuous links to a few things (thyroid disease, infections in the first few weeks of life, not getting immunizations and maybe genetics). It is mostly in very young children. Most cancers and/or disease are inherited (our genes), caused by the environment (chemicals, what we eat, where we live), radiation (sun, electronics,...) or by viruses and bacteria. We do not know what is the case for histiocytosis. Note: when we ask if a disease is 'genetic' we are really trying to ask if it is inherited or if it is genetically inherited. They problem with asking if a disease is 'genetic' is that many diseases are genetic or they are caused by problems with our genes, but they are not inherited - the problems have arisen in us, not in prior generations.Q. Does Ben have Diabetes Insipidus?
A. No. But he may someday. LCH patients tend to have the endocrine system (specifically the pituitary gland). The front part of the pituitary gland regulates how much you drink and urinate. When this system malfunctions, you drink and urinate too much. To manage this, you need to take replacement hormones for the remainder of your life. To date, there is no effective therapy to reverse DI once it has been established. DI is typically seen about a year after diagnosis of the disease. The aft part of the pituitary gland controls growth. When this malfunctions, patients are usually placed on growth hormones.Q. Is Ben likely to get Diabetes Insipidus?
A. Diabetes insipidus occurs in 5-50% of patients and is associated with the following risk factors: Skull and orbital lesions, multisystem disease and intracranial extension of osseous lesions. Ben is in the high risk group for DI.Q. What are neupogen shots? What do they do? When did your doctor feel it was warrented?
A. Neupogen is a drug or chemical they give to stimulate Neutrophil production. Neutrophils are the white blood cells that circulate in your blood and specifically fight 'blood' infections. With most chemotherapies, the immune systems takes a heavy hit. Neutrophils are easily wiped out in patients like Ben. If he did not take this 'chemokine' to stimulate Neutrophil production he would be at risk for many infection, some from his own body. We give him a shot in the leg on the doctors orders each night. We measure his blood counts twice a week and if they are low, specifically the absolute Neutrophil count or ANC, we give him the neupogen. We have to stop giving the neupogen three days before we with to attempt chemotherapy, in those three days the artificially high Neutrophil count that was propped up by the neupogen falls back to its 'true' level. Neupogen is more technically know as Granulocyte Colony Stimulating Factor (GCSF) (i.e. it stimulates the growth of the precursor cells to neutrophils which in turn turn into Neutrophils).Q. What was Ben's condition after the first 2-CdA treatment? Did it improve a lot, a little?
A. With Ben there are several ways we are measuring improvement:When Ben was on his first series of chemotherapy (Vinblastine, Prednisone & Methotrexate) we saw: 1) Tumors becoming more numerous, pronounced and larger (at a very fast rate), 2) No change in contrast levels, 3) Increased lung involvement, 4) Potential addition of kidney as an area, 5) Potential balance issues (but obviously he could not tell us), 6) Scalp lesions would really flare up and get bright red with the administration of Methotrexate and 6) a period where his eye swelled halfway shut
- Do the tumors measure smaller or the same size from one MRI scan to the next?
- Are the tumors taking up as much contrast die from one MRI scan to the next?
- Are his other areas the same or smaller (lung and kidney)?
- Are there any new areas of disease?
- Is he showing any progressive symptoms - seizure, lack of balance, lack of strength, extreme fussiness, not him self?
- Are his scalp lesions better or worse?
- Any eyelid swelling from his orbit bone lesion?
When we switched to 2-CdA after we finally realized the other chemo was not working, the first thing we saw as the scalp lesions go away. Since then his brain lesions have not gotten smaller BUT they have not gotten bigger. The latest thing is that they are not taking up contrast as well either, which the doctors say is a good thing, but it is frustrating because then it is almost impossible to measure their size.
Now, for the big issue. For some reason - which they do not understand yet - the 2-CdA works only on about 37% of the children with histiocytosis. When it work it works well, when it does not work, it does not seem to work at all. So this makes it very difficult to generalize the results of one patient to that of another.
