For most patients with classical hematology conditions, today’s treatments offer reasonable control. For a small group of patients who have exhausted every available option, CAR T-cell therapy may offer something far more durable – a one-time treatment designed to reset the immune system entirely.
What is classical hematology – and why does it matter for your patients?
Classical hematology encompasses abnormalities of the cellular and clotting components of the blood that don’t involve malignancy. Think abnormal blood counts – hemoglobin, white cells, platelets too high or too low – as well as unexplained clotting and bleeding disorders. Among the most challenging to treat are immune thrombocytopenia (ITP) and immune hemolytic anemia, autoimmune disorders in which the body makes antibodies against its own platelets or red blood cells.
“These conditions have traditionally been treated with prednisone and prednisone-like equivalents, and newer drugs have helped a lot of patients,” says hematologist Marcel Devetten, MD. But unfortunately, a small category of patients have multiple recurrent disease despite every drug known to man.”
When standard treatment isn’t enough
For patients cycling through medications with diminishing returns, the clinical picture can look deceptively stable from the outside. Dr. Devetten encourages physicians to look beyond lab values and ask patients how they’re actually living.
“There might be a misconception that patients are doing fine – but when you ask them, they’re really not doing that well,” he says. “A doctor may say, ‘As long as you’re on this medication and have an occasional transfusion, you’re okay.’ And the patient may say, ‘Yes, but I’m missing so much time at work, and I can never go on vacation with my family.’”
Signs that a patient may need more advanced evaluation include:
- Bleeding significant enough to require transfusions
- Inability to maintain normal work or family activities
- Travel restrictions due to proximity-to-care requirements
- Ongoing disease recurrence when medications are tapered
“We should really think about whether there are additional options available for any patient who is not able to live their normal life,” Dr. Devetten says.
Why CAR T-cell therapy is generating interest in classical hematology
CAR T-cell therapy has long been associated with blood cancers. Now, its success in severe rheumatologic autoimmune diseases, including scleroderma and lupus, is prompting hematologists to ask whether the same immune-resetting logic applies to refractory non-malignant blood disorders.
The premise is straightforward: If a patient’s immune system is attacking its own blood components and conventional therapies can no longer stop it, a targeted cellular therapy that fundamentally reprograms immune behavior may succeed where medications have failed. Data from Chinese research centers have shown long-term remission in patients with immune hemolytic anemia treated with CAR T-cell approaches, with many patients requiring no additional treatment.
“The hope is that this will essentially reset the immune system in such a way that it is a long-term solution,” Dr. Devetten says. “There are a ton of medications on the market right now that have been shown to be reasonably effective short-term solutions, but for most patients, at some point, either the treatment no longer works, or they try to stop it, and their disease recurs. With CAR T-cell treatment, the idea is a one-time treatment with years of benefit and no further intervention needed.”
The Nebraska Medicine role in advancing this therapy
Nebraska Medicine is participating in a combined phase 1 and phase 2 clinical trial evaluating CAR T-cell therapy for patients with refractory ITP and immune hemolytic anemia. The trial uses an allogeneic, off-the-shelf product derived from volunteer donors. Eligible patients receive a mild conditioning chemotherapy regimen followed by the infusion, and the trial is currently open to enrollment.
“Fortunately, there aren’t too many patients who are in this position where nothing works for them anymore,” Dr. Devetten says. “But once you are in that position, this could be a game changer, because once you’re there, you spend more time at the doctor’s office than going to work, or taking care of your children, or doing the things that normal people do all the time.”
Why early referral matters
For patients who may be candidates for advanced therapies or clinical trials, evaluation takes time. Workup involves extensive testing, informed consent discussions, and shared decision-making that can’t be compressed into a single visit.
“I always tell my patients, there’s a period of time which is essentially going from a first date to deciding if you want to have a long-term relationship,” Dr. Devetten says. “Don’t think that you can come here on Wednesday and start your treatment on Thursday – coming a little bit earlier in the course of disease is probably a good idea.”
A specialized resource for complex cases
Nebraska Medicine | Fred & Pamela Buffett Cancer Center is one of the few institutions in the region with physicians who dedicate themselves full-time to benign hematologic disorders. Dr. Devetten and hematologist and oncologist Alex Nester, MD, welcome referrals for patients who may benefit from a second opinion or clinical trial evaluation and are available for consultations.
“For those patients who are really struggling with their disease, this is a great place for a second opinion or a potential clinical trial,” Dr. Devetten says. “If referring physicians just want to have a quick curbside, please don’t hesitate to reach out.”
To refer a patient or request a consultation, call 402.559.5600.