Monday, May 10, 2010

Benefits of NIH Hairy Cell Leukemia Trial Participation

I've read a lot of bulletin board posts with various insights regarding participation in the NIH Trial for Newly Diagnosed HCL Patients so since I'm participating in the trial, I thought I'd offer my own.

While I have the advantage of living close to NIH, I've been in touch with many patients who have had to deal with the logistics of participating in the trial from all over the country.  Here are many of the trades people make when considering trial participation:

Trade #1:  Can I afford to travel to or stay at NIH for 8 weeks of Rituxan treatments?
         Fact: You don't have to.  Rituxan can be administered by your local doctor.  You only need to travel/stay at NIH for the first week of chemotherapy treatment.  NIH will send the Rituxan to your local doctor and coordinate the administration free of charge.

Trade #2:  My local oncologist can provide the Cladribine and Rituxan combination, so there's no need to participate in the trial.
         Fact: While local administration of the Cladribine/Rituxan combination is becoming more common,  most insurance will only cover the cost of 4 weeks of Rituxan.  By participating in the NIH trial, you'll receive up to 16 weeks (2 cycles at least 6 months apart with 8 weekly rounds per cycle) of Rituxan free of charge.  Most insurance companies view the free NIH-provided Rituxan and expected increase in remission as a major cost-savings and will cover the entire cost of local Rituxan administration (local Dr. time and facilities costs).  Since Rituxan effectivity is dependent on the size/radius of the remaining clumps of hairies, long-lasting remission and eradication is much more likely with 8 cycles per treatment than with 4.  Independent adminstration of Rituxan without monitoring minimal residual disease (MRD) in a clinical trial setting does not provide an effective means of treating the disease.  Once the trial is completed,  local oncologists will be provided with the most effective combination therapy treatment.
      
Trade #3:  The trial doesn't provide any major advantage.
          Fact:  Prior trials in Italy combining Rituxan and Cladribine resulted in a significant increase in complete remissions and elimination of minimal residual disease (MRD).  It is believed that this may increase the duration of remission, which is being studied by the NIH trial.  The NIH hyper-sensitive MRD test is able to detect 1 hairy in 1 million mononuclear cells -- 100 times more sensitive than a standard FACS.  This is a significant advantage -- allowing Rituxan to be administered at a point when it will be most effective.  This also means that Rituxan will only be administered if it is necessary.  Only NIH can perform this test!
                    Whereas most doctors aren't proactive in following their patients and wait until blood counts   indicate the need for re-treatment, Dr. Kreitman will actively study your progress and treat the earliest signs of MRD, which may possibly increase the duration of your remission (one of the hypothetical effects being studied).

Trade #4:  There are risks associated with Rituxan.  Cladribine is highly effective in most patients, so I'll do that for now and only take Rituxan later if it becomes necessary.
          Fact:  Data indicate that 40% of patients relapse after only 10 years of single-drug chemotherapy.  To prevent significant bone marrow suppression, it's desired to limit a patient to two treatments of purine analogue chemotherapy (Cladribine or Pentostatin).  The prevailing theory is that Rituxan is most effective when used in conjunction with a purine analogue so that the clumps of hairies are unclumped/diffused prior to administering it (being studied by the trial).  This allows Rituxan to destroy the individual hairy cells immediately instead of slowly peeling the outer layers of the hairy clumps (resulting in a direct additive effect vs. a percentage of the remaining load).  Don't lose this advantage by deferring Rituxan treatments until after chemotherapy is no longer viable.  One look at my ANC plots (see prior posts), and you can see I owe my overall strong response to the Cladribine/Rituxan combination.

Trade #5:  I can't afford to travel to NIH.
        Fact:  A lot first-time patients deal with the cost/benefit trade-off of travel to NIH, and rightly so.  Once accepted into the trial, NIH will cover the costs of all subsequent travel for the patient (spouses and children are not covered).  Angel Flight and the Air Charity Network offer flights to patients in financial need.

Trade #6: I can't afford accommodations while at NIH.
       Once admitted to the trial, NIH will provide free accommodations through their in-patient hospital.  While qualifying for the trial, you may need to spend two nights at a local hotel.   Current rates for the Bethesda Marriott are approximately $139/night.  You'll find that the current commercial rates are often better than the NIH negotiated rates, so ask for both.

The best way to make a fully informed decision is to gather as much information as possible.  Dr. Kreitman is always available and will quickly respond to your questions.  Email him at kreitmar@mail.nih.gov.

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