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By Nancy Lapid, Health Science Editor

Hello Health Rounds readers! Today we highlight two high profile studies presented at the ongoing American Society of Hematology (ASH) meeting in San Diego. The first involves a new bone marrow transplant approach that could help more patients with severe sickle cell disease. The other looks at a drug combination that helped people with advanced mantle cell lymphoma go significantly longer without disease progression.  

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More patients with severe sickle cell disease may be eligible for potentially curative bone marrow transplants using a new approach that is far less toxic than older methods and allows for more relatives to become donors, according to results of a study presented on Tuesday.

The usual treatment requires that the donor be a sibling who is a close genetic match, and involves intensive chemotherapy with side effects that can be difficult to tolerate, especially for patients with organ damage from the disease.

The new procedure, presented by researchers at the American Society of Hematology (ASH) meeting, uses a milder chemotherapy regimen. In addition, the donor only needs to be “half-matched” genetically to the recipient, which broadens the pool of potential donors to include parents, children, cousins, aunts, and uncles.

“This is a pragmatic approach for adults with sickle cell disease, and the results are fantastic,” study leader Dr. Adetola Kassim of Vanderbilt University Medical Center said in a statement. “It actually beat our expectations.” 

In sickle cell disease, red blood cells become misshapen, diminishing their ability to carry oxygen and causing episodes of severe, often debilitating pain as well as damage to tissues and organs that can lead to early death.

Forty-two adults with sickle cell disease and a history of stroke or reduced heart functioning, pain episodes, or frequent blood transfusions underwent the modified transplantation procedure, receiving stem cells from a family member that would produce healthy blood cells.

Two years later, 95% were alive, and 88% had functioning grafts without needing another infusion of stem cells, the researchers said.

Most participants experienced significant improvements in markers of healthy blood functioning, a reduction in pain episodes and fatigue, and improved heart and lung functioning, they added.

“This study has clearly shown that you can take selected adult patients (who are too weak to withstand the usual transplant procedure) and hopefully change the trajectory of their disease,” Kassim said.

Read more about sickle cell disease on Reuters.com

• U.S. FDA approves two gene therapies for sickle cell disease
• Vertex/CRISPR price sickle cell disease gene therapy at $2.2 mln
• Sickle cell cure will be cost-effective if health disparities considered -study

In patients with relapsed or hard-to-control mantle cell lymphoma, a combination of two oral medications from AbbVie achieved an overall remission rate of 82% and prolonged the time until the disease got worse by about 10 months, researchers said on Tuesday at the ASH meeting.

“We are very encouraged by the data from this trial… using this targeted therapy. This combination allowed us to attack the cancer cells in two ways, which made it harder for the tumor to find resistance,” study leader Dr. Michael Wang of the University of Texas M. D. Anderson Cancer Center said in a statement.

Mantle cell lymphoma is an aggressive cancer often diagnosed at an advanced stage. It is presently diagnosed in about 4,000 patients each year in the United States and is becoming more common as the population ages, Wang said.

The late-stage trial involved 267 patients who had previously received at least one prior line of therapy. One group was treated with a combination of Imbruvica, a BTK inhibitor, and Venclexta, which has a different mechanism of action known as a BCL-2 inhibitor, while the others received Imbruvica and a placebo.

With half of them followed for more than four years, the average time without disease progression was roughly 32 months with the combination versus 22.1 months for the control group.

“We look forward to longer follow-up data from this trial,” Wang said, adding that the results are “a milestone achievement for our patients with mantle cell lymphoma.”

This newsletter was edited by Bill Berkrot.

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