11 April 2023

People with a rare, precancerous blood disease benefit from a type of immunotherapy treatment, according to a new study.

Researchers at the US National Institutes of Health (NIH) found individuals with low-grade lymphomatoid granulomatosis who are treated with interferon alfa-2b can live for decades after diagnosis.

A single-centre phase 2 trial, published in Lancet Haematology, showed patients who were treated with interferon alfa-2b lived for a median of about 20 years. Previous studies reported a median survival of under two years for people with lymphomatoid granulomatosis.

Dr Christopher Melani, of the National Cancer Institute’s (NCI) Center for Cancer Research hailed the results, saying: “I think the results of this study represent a significant contribution to determining the standard-of-care treatment for this rare disease.”

Lymphomatoid granulomatosis causes an overproduction of B lymphocytes, leading to lesions in the lungs, central nervous system, skin, liver, and kidneys.

Left untreated, the disease can progress to a high-grade form, which has a poorer prognosis and can quickly turn into an aggressive and fatal B-cell lymphoma.

While chemotherapy is the standard treatment for people with high-grade disease, there is no standard treatment for low-grade disease.

Dr Wyndham Wilson, from NCI’s Center for Cancer Research, and colleagues conducted a trial of 67 people with lymphomatoid granulomatosis, 37 of whom had low-grade disease and 30 with high-grade disease.

The participants had not yet been treated for the disease, or their disease had not responded to or had returned after other treatments.

During their initial treatment, most of the patients with low-grade disease received subcutaneous injections of interferon alfa-2b three times a week in increasing doses for about one year. Most of the patients with high-grade disease were given six cycles of intravenous chemotherapy every three weeks.

Both groups improved, with the disease disappearing in 61% (27 of 44) of patients treated with interferon alfa-2b, and 47% (eight of 17) of patients treated with chemotherapy.

Some patients subsequently received crossover treatment, which was also effective. The disease disappeared in 50% (four of eight) patients treated with interferon alfa-2b after chemotherapy, and 47% (seven of 15) patients treated with chemotherapy after interferon alfa-2b.

Median overall survival was 20.6 years for patients treated initially with interferon alfa-2b and 19.8 years for patients who crossed over to receive interferon alfa-2b.

Dr Melani said: “The trial of interferon alfa-2b established that immunotherapy improves survival in patients with low-grade lymphomatoid granulomatosis.

“Now we can look into more novel immunotherapies that are easier to tolerate to see if they can improve on the efficacy of our current treatment.”


Melani C, Dowdell K, Pittaluga S, Dunleavy K, Roschewski M, Song JY, Calattini S, Kawada JI, Price DA, Chattopadhyay PK, Roederer M, Lucas AN, Steinberg SM, Jaffe ES, Cohen JI, Wilson WH. (2023) “Interferon alfa-2b in patients with low-grade lymphomatoid granulomatosis and chemotherapy with DA-EPOCH-R in patients with high-grade lymphomatoid granulomatosis: an open-label, single-centre, phase 2 trial.” Lancet Haematology, doi: 10.1016/S2352-3026(23)00029-7

Link: https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(23)00029-7/fulltext

Disclaimer: The news stories shared on this site are used as a way to inform our members and followers of updates and relevant information happening in Haematology. The BSH does not endorse the content of news items from external sources, and is not in a position to verify the findings, accuracy or the source of any studies mentioned. Any medical or drugs information is provided as an information resource only, and is not to be relied on for any diagnostic or treatment purposes.

News service provided by Englemed News.