Caution crucial for stem cell transplant in scleroderma, despite potential interest

 

SANDESTIN, FLA. – An expert called for restraint with the use of stem cell transplantation in scleroderma at the annual Congress of Clinical Rheumatology, emphasizing that the early mortality risk shows that the patient group that should be considered for the treatment is very narrow.

Results published earlier this year found a long-term benefit for myeloablative autologous stem cell transplantation, compared with cyclophosphamide, and were highly encouraging – but only in patients with severe disease (N Engl J Med. 2018;378:35-47). This might prompt patients to express interest in the treatment, many of whom are not suitable, said Janet Pope, MD, chair of rheumatology at St. Joseph’s Health Care in London, Ont.

“Your patients with scleroderma are savvy. They will be on the Internet,” she said. “They know someone who knows someone who told them about it. Most of our patients are not eligible.”

Because of mortality seen in the first year in the stem cell transplant group, the benefit over cyclophosphamide was seen only in subsequent years, Dr. Pope stressed.

Consideration of the approach is only appropriate for patients with early diffuse scleroderma who have a 50% mortality risk over 5 years, Dr. Pope said. Patients in the SCOT trial had a modified Rodnan Skin Score (mRSS) of 30, with an average forced vital capacity of 74%, and 73 of the 75 patients in the trial had lung involvement. Essentially, Dr. Pope said, these are patients with some organ involvement that could be lethal, but not such severe organ involvement that it requires a transplant.

“These are our sick patients,” she said.

The results published this year are even more definitive, Dr. Pope said, considering that they largely meshed with results out of Europe, published in 2014, that showed that the stem cell transplant benefit was not seen after the first year because of early mortality (JAMA. 2014 Jun 25;311[24]:2490-8). In that trial, a 10% mortality risk was seen in the first year in the transplant group, and then a benefit to transplant in the long term over cyclophosphamide.

 

“These are very complementary positive trials,” Dr. Pope said. “We don’t want to kill 10% of our patients who don’t have perceived high chance of mortality in the next 5 years. ... These patients are highly selected, but I think it gives our patients hope and, for some of my patients, this will be a treatment for them.”

The long-term efficacy is also encouraging in that new but less potent treatments might yield good long-term results without the early deaths, she said. “It gives an idea and hope, and it helps us to understand that maybe with immune modulation, without the nuclear blast of stem cell transplant, maybe we can do better for our patients.”

Dr. Pope reported relevant financial relationships with Actelion, Bayer, Merck, Bristol-Myers Squibb, and Roche.

 

SANDESTIN, FLA. – An expert called for restraint with the use of stem cell transplantation in scleroderma at the annual Congress of Clinical Rheumatology, emphasizing that the early mortality risk shows that the patient group that should be considered for the treatment is very narrow.

Results published earlier this year found a long-term benefit for myeloablative autologous stem cell transplantation, compared with cyclophosphamide, and were highly encouraging – but only in patients with severe disease (N Engl J Med. 2018;378:35-47). This might prompt patients to express interest in the treatment, many of whom are not suitable, said Janet Pope, MD, chair of rheumatology at St. Joseph’s Health Care in London, Ont.

“Your patients with scleroderma are savvy. They will be on the Internet,” she said. “They know someone who knows someone who told them about it. Most of our patients are not eligible.”

Because of mortality seen in the first year in the stem cell transplant group, the benefit over cyclophosphamide was seen only in subsequent years, Dr. Pope stressed.

Consideration of the approach is only appropriate for patients with early diffuse scleroderma who have a 50% mortality risk over 5 years, Dr. Pope said. Patients in the SCOT trial had a modified Rodnan Skin Score (mRSS) of 30, with an average forced vital capacity of 74%, and 73 of the 75 patients in the trial had lung involvement. Essentially, Dr. Pope said, these are patients with some organ involvement that could be lethal, but not such severe organ involvement that it requires a transplant.

“These are our sick patients,” she said.

The results published this year are even more definitive, Dr. Pope said, considering that they largely meshed with results out of Europe, published in 2014, that showed that the stem cell transplant benefit was not seen after the first year because of early mortality (JAMA. 2014 Jun 25;311[24]:2490-8). In that trial, a 10% mortality risk was seen in the first year in the transplant group, and then a benefit to transplant in the long term over cyclophosphamide.

 

“These are very complementary positive trials,” Dr. Pope said. “We don’t want to kill 10% of our patients who don’t have perceived high chance of mortality in the next 5 years. ... These patients are highly selected, but I think it gives our patients hope and, for some of my patients, this will be a treatment for them.”

The long-term efficacy is also encouraging in that new but less potent treatments might yield good long-term results without the early deaths, she said. “It gives an idea and hope, and it helps us to understand that maybe with immune modulation, without the nuclear blast of stem cell transplant, maybe we can do better for our patients.”

Dr. Pope reported relevant financial relationships with Actelion, Bayer, Merck, Bristol-Myers Squibb, and Roche.

 

SANDESTIN, FLA. – An expert called for restraint with the use of stem cell transplantation in scleroderma at the annual Congress of Clinical Rheumatology, emphasizing that the early mortality risk shows that the patient group that should be considered for the treatment is very narrow.

Results published earlier this year found a long-term benefit for myeloablative autologous stem cell transplantation, compared with cyclophosphamide, and were highly encouraging – but only in patients with severe disease (N Engl J Med. 2018;378:35-47). This might prompt patients to express interest in the treatment, many of whom are not suitable, said Janet Pope, MD, chair of rheumatology at St. Joseph’s Health Care in London, Ont.

“Your patients with scleroderma are savvy. They will be on the Internet,” she said. “They know someone who knows someone who told them about it. Most of our patients are not eligible.”

Because of mortality seen in the first year in the stem cell transplant group, the benefit over cyclophosphamide was seen only in subsequent years, Dr. Pope stressed.

Consideration of the approach is only appropriate for patients with early diffuse scleroderma who have a 50% mortality risk over 5 years, Dr. Pope said. Patients in the SCOT trial had a modified Rodnan Skin Score (mRSS) of 30, with an average forced vital capacity of 74%, and 73 of the 75 patients in the trial had lung involvement. Essentially, Dr. Pope said, these are patients with some organ involvement that could be lethal, but not such severe organ involvement that it requires a transplant.

“These are our sick patients,” she said.

The results published this year are even more definitive, Dr. Pope said, considering that they largely meshed with results out of Europe, published in 2014, that showed that the stem cell transplant benefit was not seen after the first year because of early mortality (JAMA. 2014 Jun 25;311[24]:2490-8). In that trial, a 10% mortality risk was seen in the first year in the transplant group, and then a benefit to transplant in the long term over cyclophosphamide.

 

“These are very complementary positive trials,” Dr. Pope said. “We don’t want to kill 10% of our patients who don’t have perceived high chance of mortality in the next 5 years. ... These patients are highly selected, but I think it gives our patients hope and, for some of my patients, this will be a treatment for them.”

The long-term efficacy is also encouraging in that new but less potent treatments might yield good long-term results without the early deaths, she said. “It gives an idea and hope, and it helps us to understand that maybe with immune modulation, without the nuclear blast of stem cell transplant, maybe we can do better for our patients.”

Dr. Pope reported relevant financial relationships with Actelion, Bayer, Merck, Bristol-Myers Squibb, and Roche.