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MUD Transplants at the NCI

Date: January 17, 2008

by Chaya Venkat

Brand New Protocol Makes Its Debut

Related Articles:
Transplants Almost Free;
Only Real Cure;
Matcing Made Simple;
Catch 22 - The Transplant Minefield;
What Say You Dr. Expert?;
Primer on SCTs.


Hope Is a Wonderful Thing

Sometimes, the light at the end of the tunnel is not a train rushing at you at breakneck speed, sometimes it is good news arriving just in the nick of time. This article reviews a very important clinical trial at the NCI (the U. S. National Cancer Institute). That’s right, your tax dollars at work and actually doing you some good! Who would have thunk it, this program may actually offer options to some of our patients that faced an otherwise bleak future. Read on, I promise you will be glad you did. After all, this NCI MUD transplant program offers as close to the proverbial free lunch as you can get, and still be legal.

Mini-allo MUD Transplants: Logic, Roadblocks and Finances

If you are a CLL patient with aggressive disease, and you are not likely to run out the clock by using conventional therapy, you should know by now that the only way of curing this incurable cancer is by means of a hematopoietic (blood) stem cell transplant. If this is news to you, please read some of the review articles we have on this subject. The life you save may be your own! If you know all about this business from reading our prior articles, pardon me for being a bore and going over the details again. Our audience keeps growing, there are always new members facing these issues for the first time, and as I say many times, the devil is in the details.

Hematopoietic stem cell transplants (HSCT) have been around for a quite a while. The full myeloablative, take-no-prisoners variety of transplants, where patients are subjected to very heavy duty pre-conditioning therapy (including high dose full body radiation) have been practiced for more than a couple of decades. If you are a young patient, fit as a fiddle, and able to compete with the likes of Lance Armstrong, this is no big deal. But for us merely mortal beings getting a little long in the tooth and not exactly athletic, very often the full myeloablative transplants are far too aggressive. The treatment related mortality (TRM, or what I like to call death-by-therapy) of myeloablative transplants can be pretty daunting. That is why they have strict age and fitness requirements before they enroll patients in full myeloablative transplant protocols.

Mini-allo (also called Reduced Intensity Conditioning (RIC) or non-myeloablative) stem cell transplants are a relatively new innovation. In this approach, the preconditioning is not anywhere as aggressive. Most often it is no more aggressive than standard chemotherapy combinations used to control CLL. This approach became possible when researchers discovered that it is not necessary to try and kill every CLL cell in the body prior to introducing the patient to his/her new immune system. The breakthrough happened when they discovered the graft-versus-leukemia (GVL) effect (also called graft-versus-cancer or graft-versus-tumor effects) where the newly engrafted immune system goes out hunting and kills the remaining cancer cells in the body. A new era dawned, making it possible for older and less fit patients to have transplants that can potentially cure them of their otherwise incurable cancer. Please understand all through this article we are talking of allogeneic transplants, where the patients own cancer corrupted immune system is replaced with a healthy one from a matched donor. This is very different from autologous stem cell transplants, where the patient’s own stem cells are used. Auto transplants are not considered to have the ability to cure CLL.

Getting a stem cell transplant is not a simple matter. For starters, there is the issue of finding a suitable donor. Sibling donors were the preferred donors, by far, until just recently. As you would expect, much of the original work with both myeloablative and mini-allo transplants were done with well matched sibling donors. It makes sense, your brothers and sisters have the highest probability of being a good match. While there is no guarantee that any particular sibling will be a good match, the larger the number of brothers and sisters you have, the better the chances that you will find a good match in one or more of them. There is also this little thing called motivation – your siblings are more likely to want to save your life by agreeing to donate some of their stem cells. If that is not the case in your family, perhaps you should review your choice of birthday and Christmas presents to your siblings in the years past? Just kidding.

How about patients without siblings, or at least siblings that do not match? For these folks, matched unrelated donors (MUD) are the next best choice. With the exception of ethnic minorities, it is generally feasible to find good matches in the donor registries. Most of the good transplant protocols require a “perfect” match of 10 out of 10 HLA markers, tested at the high resolution level. Finding a good match is not a simple matter. If you used one of the “lay public” matching engines on internet blood registry sites and came up with dozens of matches, it is not safe to assume you are guaranteed a good match. First, most of these lay search engines report matches at a two digit low resolution level – not good enough when rubber meets the road. Second, most of these engines try to match only 6 HLA antigens, where as the state of the art requirement is 10 HLA match. Last but not least, just because there is this dude with the perfect 10 point high resolution match for you on the donor registry does not mean you are home free. The dude may have changed his mind, picked up hepatitis infection or worse since he signed up to be a donor, or has moved and left no forwarding address. Finding a good MUD match that actually pans out in real life is serious business, needs to be done by a competent institution, and it often costs a lot of money. The more exotic your ethnicity, the harder the job of finding you a good match, and the more it will cost. When we went through this exhaustive search process in trying to find a match for my husband PC, it cost our insurance company in the neighborhood of $30K – and at the end of it, we came up empty. There was no MUD match available for him. Bummer.

Mini-allo transplants work best when the patient goes in with bare minimum number of cancer cells in his body. This makes sense, it gives the new graft coming in a chance to settle down, marshal its own troops and get its act together before the remnants of the cancer cells grow out of control. We have discussed in prior articles the importance of timing in deciding when to initiate the transplant decision. If you wait too long, it may be hard to get that clean remission just before the transplant, increasing your risk of relapse after the transplant; and your body may be the worse for wear if you had to use every bullet known to man by way of chemotherapy, making you more vulnerable to death-by-therapy (TRM). It is far better if you go in healthy, with few or no complicating health conditions.

As you might have gathered by now, money is a very real issue in getting a stem cell transplant. The full cost of a transplant can vary a great deal, depending upon the institution, level of complexity and long term complications. The low ball number is around $500K, but I have heard scary reports that they can be as much as a couple of million dollars. Insurance companies don’t exactly like paying out that kind of cash, and some of the less reputable ones will not cover transplants because they are “not proven”. The more up-front policies have outright exclusions where transplants are concerned, or have lifetime maximum caps on how much they pay, thereby effectively locking out patients from such expensive procedures. Don’t even get me started on all the millions of people in this country that don’t have any kind of medical insurance.

Here then is the cheat sheet of transplant roadblocks:

  1. Deciding to get a transplant while you are still healthy enough to survive the process. This is by no means an easy decision, and not one to be made for trivial reasons.
  2. Getting a good remission ahead of the transplant, thereby giving the new graft a good chance of winning the battle. Waiting too long may make this tough to achieve.
  3. Finding a well matched MUD donor (assuming you do not have a well matched sibling) – luck, money, persistence and a knowledgeable institution’s help are needed!
  4. Paying for the whole darn thing, either through insurance or out of your own pocket. It is not cheap, by any stretch of the imagination, and don’t assume your insurance company will be more than delighted to help you.
  5. Trying to minimize the nasty side effects of graft-versus-host disease (GVHD)

I told you this is a good news report you will want to read and bookmark. There is a state of the art, mini-allo MUD transplant clinical trial protocol at one of our premier research institutions, CLL patients are eligible, and here is the clincher – almost all of it is free of charge. I am not kidding folks. They will even pay for the donor registry search to find you a good MUD match! The protocol is open to all USA residents, as well as foreigners who are in this country with a valid visa. 20-25 patients will be enrolled. This protocol has just opened, and they already have a few patients enrolled. My guess is the remaining slots will not stay open for too long. The inclusion criteria are for a variety of blood cancers, not just CLL. But between you and me, I would not be sorry if most of the slots are taken up by CLL patients. Call me selfish why don’t you.

The NCI Mini-allo MUD Transplant Protocol

The link to the National Cancer Institute’s protocol that is of interest to us is given below. Please read it carefully. It has lots of important details, inclusion criteria, contact information etc. I spent a good two hours on the phone with Dr. Michael Bishop, the Principal Investigator in charge of this clinical trial. Dr. Bishop was also kind enough to send me several of his published articles as well as backup information. My layperson review of their protocol is an attempt to make some of the technical information more understandable to patients and their families. Obviously, the trial protocol has the final word on all of this. Write to us if you want more detailed information, or contact the NCI folks directly.

NCI Clinical Trial Protocol NCI-07-C-0195

Your best bet to see if you are eligible is to read their inclusion criteria carefully. As is common, the protocol requires that you must have had fludarabine therapy (either as single agent or in a combo) in your past, and relapsed. But here is a detail that I got from my discussion with Dr. Bishop. If you have autoimmune disease such as AIHA (autoimmune hemolytic anemia) and therefore fludarabine is not considered appropriate for you, this automatically satisfies the condition. Contraindication of fludarabine is equivalent to fludarabine refractory, for the purposes of this trial inclusion.

As we discussed above, a successful transplant can be divided into roughly three parts:

  1. Getting a good remission ahead of the transplant itself. This is also called “Induction chemotherapy” and could take months.
  2. The transplant itself, consisting of pre-conditioning just days ahead of giving the patient the new stem cells
  3. Managing the post transplant complications, making sure the precious new graft is not rejected, preventing infections and CLL relapse, and reducing the complications of GVHD while encouraging curative powers of GVL.

Most transplant centers handle only the second and third items above. If you go in for a consultation, the transplanter is likely to tell you to go back home, get yourself a good remission by whatever method your CLL guy recommends, then come back when you are ready for the transplant. If you have become chemo-insensitive, your disease no longer responds to chemotherapy, many transplant programs will turn you away. Not so the NCI protocol. This protocol is far more thorough, it addresses all three of these parts, including getting you into a good remission to begin with. And it will accept patients who are refractory to most standard CLL chemotherapy, even those that have morphed into the dreaded Richter’s transformation.

Step 1: Induction Chemotherapy: Getting a Good Remission Prior to Transplant

Most patients need pretty heavy duty therapy if they are going to get a clean remission ahead of the transplant. It makes sense, if you are able to get a clean remission with nothing more than plain vanilla Rituxan or green tea, for heaven’s sake, you are not a good candidate for a transplant! Why would you want to take all that risk, if you are able to get by on relatively benign ways of controlling your CLL? But most of us know that aggressive CLL will eventually need more than single agent monoclonal therapy to control it, and sometimes it is hard to get a good remission even with chemoimmunotherapy combinations such as RF and FCR.

Majority of patients facing a transplant have large lymph nodes. Poor prognostic indicators such as 11q (ATM) and 17p (p53) deletions are typically accompanied by swollen lymph nodes, and CLL cells hiding in these swollen glands are hard to kill. (Patients with Trisomy 12 are also often seen with massive nodes). From a simplistic view, these poor prognostic and potentially later stage CLL patients are more like lymphoma patients than leukemia patients, in the sense that the bulk of their tumor cells are not swimming around in the blood, most of them are safely tucked away in swollen lymph nodes, spleen, bone marrow etc. It should come as no surprise to you that many of these patients have to resort to combinations more familiar to lymphoma guys, such as CHOP and R-CHOP. (R: Rituxan; C: cyclophosphamide; H: doxorubicin (“adriamycin”); O: vincristine; P: prednisone)

The NCI protocol has an interesting approach to getting all sorts of patients into a good remission ahead of the transplant. It is important to remember this program is open to many different sorts of blood cancer patients, and CLL is grouped with a bunch of other B-cell cancers. The logic seems to be to hit the cancer from many different angles, give it fewer chances of using an escape hatch and slinking by. Their chemo cocktail uses 7 different drugs: EPOCH –FR. You know most of these drugs already. It might be easier to understand this combination if you think of it as E-CHOP-RF. We described CHOP above, and it is pretty standard therapy for lymphoma patients as well as some leukemia patients; you know RF from our many articles on the subject of this particular combination; the only new one here is E: etoposide. As you can see from the first abstract below, EPOCH-F was able to get a majority of chemo-refractory lymphoma patients into sufficient remission that they could go ahead with the transplant. It should come as no surprise to most of our readers that adding Rituxan improved the process, as shown in the second abstract. The cheat-sheet version of the induction chemotherapy is as follows:

EPOCH-F/R (for patients with Hodgkin lymphoma, non-Hodgkin lymphoma, chronic lymphocytic leukemia, prolymphocytic leukemia, or multiple myeloma):


Biol Blood Marrow Transplant. 2003 Mar;9(3):162-9.

Establishment of early donor engraftment after reduced-intensity allogeneic hematopoietic stem cell transplantation to potentiate the graft-versus-lymphoma effect against refractory lymphomas.

Bishop MR, Hou JW, Wilson WH, Steinberg SM, Odom J, Castro K, Kasten-Sportes C, Gea-Banacloche J, Marchigiani D, Gress R, Fowler DH.

Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892

Reduced-intensity allogeneic hematopoietic stem cell transplantation (alloHSCT), which typically results in mixed chimerism initially after transplantation, has had limited efficacy in chemotherapy-refractory lymphomas. We hypothesized that the rapid establishment of complete donor chimerism would potentiate a graft-versus-lymphoma effect. Fifteen patients with chemotherapy-refractory lymphoma initially received induction with a conventional chemotherapy regimen (etoposide, prednisone, vincristine, cyclophosphamide, adriamycin, fludarabine [EPOCH-F]) to deplete host T cells and provide disease control prior to alloHSCT. Patients then received conditioning with fludarabine and cyclophosphamide followed by alloHSCT from HLA-matched siblings. Graft-versus-host disease prophylaxis consisted of cyclosporine alone. EPOCH-F resulted in 73% of patients having partial responses or stable disease. EPOCH-F depleted host CD4(+) T cells from a median of 235 cells/microL to 56 cells/microL. Fourteen patients underwent alloHSCT, and all had >95% donor engraftment by day 14 after transplantation. The incidence of Grade II to III acute graft-versus-host disease was 71%. There were two therapy-related deaths. There were 8 partial responses and 3 complete responses (CRs) at day 28. Five additional CRs were observed at day 100 without withdrawal of cyclosporine or donor lymphocyte infusion. The rate of CRs for all 15 patients was 60%. The 1-year progression-free survival rate from time of study entry is 67% with only 1 relapse among 9 CRs. At a median potential follow-up of 28 months, the overall survival rate is 53%. These data demonstrate that a potent and durable graft-versus-lymphoma effect can occur against chemotherapy-refractory lymphomas and suggest that this effect may be associated with rapid, complete donor chimerism after reduced-intensity alloHSCT.

Copyright 2003 American Society for Blood and Marrow Transplantation

PMID: 12652466

Semin Oncol. 2002 Feb;29(1 Suppl 2):41-7.

The role of rituximab and chemotherapy in aggressive B-cell lymphoma: a preliminary report of dose-adjusted EPOCH-R.

Wilson WH, Gutierrez M, O'Connor P, Frankel S, Jaffe E, Chabner BA, Grossbard ML.

Center for Cancer Research, National Cancer Institute, Bethesda, MD 20892

Accumulating evidence suggests that the ability to activate apoptotic pathways may be an important determinant of chemotherapy sensitivity and presents a potentially important new therapeutic strategy. Monoclonal antibodies against the CD20 antigen directly induce apoptosis and may serve to modulate the threshold for chemotherapy-induced apoptosis. Rituximab (Rituxan; Genentech, Inc, South San Francisco, CA, and IDEC Pharmaceuticals, San Diego, CA), a monoclonal antibody against CD20, was combined with dose-adjusted EPOCH (infusional etoposide/vincristine/doxorubicin/bolus cyclophosphamide/prednisone) chemotherapy and tested in 38 untreated or relapsed poor-prognosis aggressive lymphomas. Twenty-three patients were untreated. Of these patients, all had large B-cell histologies, a median age of 52 years, Eastern Cooperative Oncology Group performance status > or = 2 in 30%, and high-intermediate or high International Prognostic Index scores in 61%. Fifteen patients had relapsed or refractory lymphomas. These patients had received a median of two (range, one to four) prior regimens, 67% had aggressive histologies, and 60% had high-intermediate or high International Prognostic Index scores. Complete remissions were achieved in 85% and 64% of untreated and previously treated patients, respectively; additionally 42% of patients with disease refractory before therapy achieved complete remission. At a median follow-up of 12 months, progression-free and overall survival in the previously untreated group was 85% and 79%, respectively, and no patient in complete remission has relapsed. These results suggest that rituximab may modulate the sensitivity of B-cell lymphomas to chemotherapy.

PMID: 11842388

Step 2: Preconditioning Ahead of the Transplant

Even if you are in perfect remission going into a transplant, you will have to get some pre-conditioning treatment ahead of getting the stem cells from your donor. The reason for it is very simple. If you have plenty of your own T-cells (“host T-cells”) going into the transplant, these defenders of your body will see the new graft coming in as the “enemy” and kill it. When this happens, it is called graft rejection and it is not at all something you want to happen. When they used to do full myeloablative transplants, where heavy duty pre-conditioning was used, there was little chance that host T-cells survived and therefore the risk of the graft getting killed was very small. But with the advent of kinder and gentler “mini-allo” transplant protocols, graft rejection has become a more significant issue. Bottom line, it is important that the host is properly prepared ahead of the transplant. The normal logic of wanting to preserve T-cells and therefore a strong immune system does not work in the transplant situation. What you want is the exact opposite. You have decided the immune system you were born with is no good, and it makes sense to throw the bums out, all of them, including the T-cells, before you introduce your body to its new immune system.

Most of the preconditioning regimens in mini-allo transplants use fludarabine, cyclophosphamide and full body radiation. The radiation used in min-allo transplants is much, much less than that used in full myeloablative transplants, usually ‘only’ 200cGY as opposed to several thousand cGY used in full myeloablative procedures. Most mini-allo transplant preconditioning also calls for something called ATG (“anti-thymocyte globulin”). This is a protein obtained for rabbits or horses, and its sole purpose is to kill T-cells.

The NCI protocol we are discussing here differs in that their preconditioning regimen does not use any radiation or ATG. That is because the EPOCH-RF combination used in the Step 1 induction therapy takes care of the T-cells, makes sure there are few host T-cells left over to hassle the graft coming in. In other words, EPOCH-RF differs from full myeloablative procedures in that it is better tolerated and much less toxic, but it also differs from more conventional mini-allo protocols in that it does not need back up of radiation or ATG in the preconditioning, in order to get rid of host T-cells just prior to transplant. As the abstract below points out, host T-cells play a very destructive role in preventing successful engraftment of the new immune system, and that in turn plays havoc with disease control. Dr. Bishop tells me that in a prior version of this protocol with sibling donors, every single patient engrafted and there was zero graft rejection.


Biol Blood Marrow Transplant. 2007 Sep;13(9):1022-30.

Host T cells affect donor T cell engraftment and graft-versus-host disease after reduced-intensity hematopoietic stem cell transplantation.

Hardy NM, Hakim F, Steinberg SM, Krumlauf M, Cvitkovic R, Babb R, Odom J, Fowler DH, Gress RE, Bishop MR.

Experimental Transplantation and Immunology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892

Mixed chimerism in the T cell compartment (MCT) after reduced-intensity stem cell transplantation (RIST) may influence immune repopulation with alloreactive donor T cells. We examined effects of host T cell numbers on donor T cell engraftment and recovery and on acute graft-versus-host disease (aGVHD) in a relatively homogeneous patient population with respect to residual host T cells through quantified immune depletion prior to RIST and to donor T cells by setting the allograft T cell dose of 1x10(5) CD3+ cells/kg. In this setting, 2 patterns of early donor T cell engraftment could be distinguished by day +42: (1) early and complete donor chimerism in the T cell compartment (FDCT) and (2) persistent MCT. FDCT was associated with lower residual host CD8+ T cell counts prior to transplant and aGVHD. With persistent MCT, subsequent development of aGVHD could be predicted by the direction of change in T cell donor chimerism after donor lymphocyte infusion, and no aGVHD occurred until FDCT was established. MCT did not affect recovery of donor T cell counts. These observations suggest that the relative number and alloreactivity of donor and host T cells are more important than the absolute allograft T cell dose in determining donor engraftment and aGVHD after RIST.

PMID: 17697964

Step 3: Post Transplant Issues

Handling post transplant issues is what separates men from boys, as far as transplant expertise is concerned. We have already discussed complications such as graft rejection (if there are too many host T-cells around and they attack the new graft coming in), CLL relapse (if the patient goes in with too much tumor load and the graft is not able to control it via graft-versus-leukemia effect), “TRM” or treatment related mortality (of particular importance to patients with other serious health problems, such as heart, lungs, liver or kidney problems). Graft-versus-host-disease (GVHD) is the other biggie. Think of GVHD as the evil twin of the beloved GVL. GVL is where the new immune system does what it is supposed to do, kill the remaining leukemia cells. GVHD is when it does what you don’t want it to do, attack perfectly innocents parts of your body. GVHD can cause damage to mucosal linings of your GI tract and mouth (think diarrhea and oral mucositis), significant skin problems, even life threatening organ damage. Unless it is appropriately controlled, GVHD can be a major factor in quality of life after transplant, it can even be the cause of death.

Both GVL and GVHD depend on the activity of graft T-cells. The art of the transplanter comes into play in allowing sufficient level of GVL to control the cancer, but putting the brakes on undesirable GVHD. In other words, a big chunk of a successful transplant depends on getting the new graft T-cells to do what we want them to do (GVL), while keeping a close control of them to make sure they don’t go “postal” on us in terms of GVHD. Standard approach to controlling GVHD is immune suppressing drugs such as prednisolone, tacrolimus, sirolimus, methotrexate etc. These drugs are used in various combinations by majority of transplant protocols. Each of these drugs has systemic immune suppressing activity, but the major function that is desired is to control the graft T-cells.

The research aspects of the NCI protocol focuses on two distinctly different approaches to controlling GVHD. Patients enrolled in this study will be randomized into two groups. The first group will get the standard combination of tacrolimus, sirolimus and methotrexate (“TMS”, popularized in institutions such as Dana Farber). The second group will get alemtuzumab “Campath”, and cyclosporine (CA). The details are given below:

TMS Group:
CA Group:

We have discussed the role of Campath therapy in many prior articles on our website. Its major asset in treating CLL is that it does a terrific job of killing CLL cells, since they carry the CD52 marker targeted by Campath. But the side effect of Campath therapy that gives us pause in using it to treat CLL is that it is also equally good at killing T-cells, and is therefore immune suppressive. Here is a case of turning conventional logic on its head: since Campath is so good at killing T-cells, it is a good way of keeping close tabs on graft T-cells when they get a little out of control, start attacking healthy tissues of the body and causing unacceptable levels of GVHD. The fact that Campath is also a good way of controlling any remaining traces of CLL is a good thing too. In any case, the whole point of this clinical trial is to see which of these two groups of patients fare better in terms of GVHD.

Contact Information

I have provided below the contact information for this important clinical trial. A word to the wise: get on the honk right away if you are interested, I doubt this trial will be recruiting for very long. At the very least, it does not hurt to find out if you are eligible, and if you are, to see if they can find you a well matched MUD in the donor registries. Remember, finding a good MUD match is neither a simple process nor cheap on the wallet, and this is something that the NCI will do free of charge for eligible patients.

Pilot Trial of Targeted Immune-Depleting Chemotherapy and Reduced-Intensity Allogeneic Hematopoietic Stem Cell Transplantation Using HLA-Matched Unrelated Donors and Utilizing Two Graft-versus-Host Disease Prophylaxis Regimens for the Treatment of Leukemias, Lymphomas, and Pre-malignant Blood Disorders



Michael Bishop, M.D.
Principal Investigator
Phone: 301-435-2764

Steven Pavletic, M.D., M.S.
Protocol Chair
Phone: 301-402-4899

Referral Contacts


Mike Krumlauf, R.N., B.S.N., O.C.N.
Research Nurse
Phone: 301-594-2056
Fax: 301-435-6830

Susan Michaud, R.N., B.S.N., O.C.N.
Research Nurse
Phone: 301-594-5525
Fax: 301-435-6830

Tiffany design


Once before, we wrote about a transplant program at the National Institutes of Health (see Stem Cell Transplants - Almost Free of Charge). A lot of our members read the article and were deeply interested in this option. Unfortunately, that clinical trial protocol was restricted to patients who had the luxury of a matched sibling donor for their stem cell transplant. A lot of our members who were interested in stem cell transplants were ruled out because they did not have a well matched brother or sister.

This new clinical trial we have reviewed in this article levels the playing field, since it is open to folks without sibling donors but are fortunate enough to find a matched unrelated donor. (And we are working hard to find an equivalent program for patients who have neither sibling nor adult MUD matches, and therefore have to depend on cord blood transplants).

Money makes the world go around, my friends. Unless you live in a cave and don’t go out much, you must be aware of the wide ranging discussions going on in this country on the state of our health care, the unacceptably large percentage of our citizens that do not have adequate health insurance. Stem cell transplants cost money. Lots of it. And since the process is still something of an art rather than cookbook technology, it really helps if you get your transplant done at a place where they know what they are doing. I have heard from too many of our members whose health insurance plans look fine at first blush, stem cell transplants are covered. The devil is in the details. Sometimes these plans cover only a percentage of the costs. It is a big deal even if you have to cover “only” 20% of the cost, if we are talking about a million dollar procedure. I don’t know about you, but that is real money where I come from. Another popular hurdle thrown at us is that the transplant cost is covered, but only if it is done at a local hospital or transplant center that is just getting geared up to do these complicated procedures. You are better off if you can vote with your feet, go someplace where they have all the different fields of expertise needed to manage the process. This is what the NCI FAQ had to say about the costs of this trial:

There is no charge for medical care received at the NIH Clinical Center. Study participants will be responsible for travel costs for their initial screening visits. Once participants are enrolled in this trial, NCI will pay for the transportation costs for all subsequent trial-related visits for participants who do not live in the local area. In addition, these participants will receive a small per diem for food and lodging expenses if they are being treated as outpatients.

Can you think of a more generous arrangement as far as money is concerned? I can’t.

There is, however, no doubt that this and just about every other transplant protocol requires substantial commitment of your time. Most if not all of them also require and strongly recommend that you need to have a full time care-giver with you. This is not a trivial requirement, you are talking about putting your life on hold for several months. But the good news is that you will actually have a life to put on hold – let us not forget an allogeneic transplant is still the only therapy of proven ability to cure this otherwise incurable cancer. This is what the NCI trial requires by way of physical stay at Bethesda, MD.

The trial would require a minimum stay in the Washington, D.C., area of a minimum of 6 months. This stay could be more if complications should occur. Approximately a third of this time occurs as inpatient. The other time is spent as an outpatient with clinic visits occurring 1–3 times a week monitoring patients for complications which may occur after transplantation (e.g., GVHD or infections).

The NCI covers some of the costs of the stay with a small per diem, but I doubt it will cover all the costs of a two person stay in the area. This is something to check out if you are truly serious about participating in this clinical trial. It may be possible for us to get our community mobilized, see if we can find locally resident members that will play host for part of the time, or make low cost rentals available. I am always amazed at the generosity of people when they are approached right. By the way, PC and I visited the Bethesda area when we were invited to make a presentation at the NIH, we found it to be a lively and happening place. Not that you would be doing too much sight-seeing or dining out as you get your immune system replaced, but what the heck, it beats being in the boonies for 6 months. Let me emphasize, this trial is open to non-USA citizens as well. You just have to be here in this country on a valid visa. Independent of your political persuasion in this charged US election year, CLL is a disease that does not respect citizenship or national boundaries. CLL Topics is proud to speak for all of our members, worldwide, and we hope you share our sense of solidarity.

Last but by no means least, I want you to spend a little time to think about the altruism behind patient participation in clinical trials. Most of us want to be good people – we want our lives to mean something positive at the end of it all. We want to take care of our family and friends, we want them to know we care about them. Well, participating in well designed clinical trials is the best way to take care of our extended family of CLL patients. If you are thinking of a stem cell transplant for taking care of your aggressive CLL, why not do it in the context of a well designed clinical trial? You come out ahead by getting the procedure at a top rated expert center, you cant get a better deal on the money side of the equation, and researchers learn more about how best to control GVHD in the transplant setting. Everyone wins.

If you decide this option may be a good one for you, I suggest you don’t dither for too long. And if you do decide to contact Dr. Bishop or the other contact individuals listed above, please don’t be shy about telling them you come from CLL Topics. A lone patient is just one voice in the wilderness, easily lost or dismissed. As a cohesive patient community we have more impact and our reach is much greater. Would you have known about either this clinical trial or the earlier sibling donor program at the NCI / NIH without patient advocacy of CLL Topics bringing you the information? If this means our CLL guys “hijack” this trial and more of them get to participate in it as compared to the other blood cancer patients without equivalent advocacy programs, well, I am not going to worry about it. I am not Mother Theresa, I am just a CLL patient spouse and CLL patient advocate – and proud of it.




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oriental pond



Topic: Transplants

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