Bone marrow transplant may be effective for PNH after, with cancer

People with PNH are at increased risk for myeloid neoplasms

Marisa Wexler MS avatar

by Marisa Wexler MS |

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A bone marrow transplant may be an effective treatment for some people with bone marrow cancer who develop subsequent or co-occurring paroxysmal nocturnal hemoglobinuria (PNH), a study indicates.

The study, “Haemolytic paroxysmal nocturnal haemoglobinuria in patients with myeloid neoplasms: A rare association with specific therapeutic implications,” was published in the British Journal of Haematology.

People with PNH are at an increased risk for myeloid neoplasms (MN), or cancers of the bone marrow. It’s much rarer, however, for someone with established MN to develop PNH, or for both diseases to be diagnosed at the same time.

In this study, scientists in France reviewed data from patients at their specialty center and emailed more than 100 other specialists practicing throughout the country.

“The purpose … was to describe the disease characteristics and clinical course of patients with a concurrent diagnosis of PNH and MN, without evidence of pre-existing PNH,” they wrote.

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The researchers identified 20 patients, including 11 diagnosed with MN and PNH at the same time, and nine who developed PNH months or years after being diagnosed with MN.

All showed signs of hemolysis, or blood cell destruction, a characteristic sign of PNH, and most required regular blood transfusions. Thrombosis (dangerous blood clotting events) occurred in half of them.

Thirteen patients were treated with Soliris (eculizumab), an approved PNH therapy. All remained dependent on blood transfusions despite Soliris. Researchers noted none had thrombosis during the Soliris treatment, though two had clotting events after stopping it.

“[Soliris] therapy in eligible patients also did not show any obvious benefit on transfusion requirements. Nevertheless, no recurrence of a thrombotic event was observed in [Soliris] therapy, which could support its use to prevent thrombosis in these high-risk patients,” the researchers wrote.

Half the patients received treatment with MN-specific therapies, such as azacytidine or ruxolitinib, though these were generally not effective at easing hemolysis or reducing the need for blood transfusions.

“MN treatments were largely inefficient on transfusion needs, and response to treatment might then not be effectively assessed following current standards,” wrote the scientists, who noted the median survival time among the patients was more than seven years after the MN diagnosis, “suggesting that MN, in our cohort, do not exhibit an aggressive [behavior].”

Five patients received a bone marrow transplant (also known as an hematopoietic stem cell transplant, or HSCT). At the latest follow-up, four of them were still alive without requiring blood transfusions.

“This suggests that HSCT should be proposed for fit patients based on MN disease risk and that thrombosis prevention, with or without [Soliris], might be a key point of pre-transplant therapeutic management,” the researchers wrote.