Safety of Autologous Hematopoietic Stem Cell Transplants (HSCT) in Systemic Sclerosis

For those of you interested in autologous stem cell transplants, this paper was just published open access in “Clinical Rheumatology”: https://link.springer.com/article/10.1007/s10067-017-3954-5. Autologous HSCT may be an appropriate treatment option to consider in certain cases of rapidly progressing disease, but this is a reminder that this is still a high-risk procedure. Here is the abstract:

“Autologous hematopoietic stem cell transplantation (AHSCT) is thought to be effective therapeutic approach in patients with poor prognosis systemic sclerosis; however, the toxicity remains a challenge. Between years 2003 and 2016, we enrolled 18 patients with systemic sclerosis at median age at transplant of 52 years (range 24–68). The median duration of disease before AHSCT was 14 months (range 2–85). Peripheral blood stem cells were mobilized with cyclophosphamide (CY) and granulocyte colony-stimulating factor. Conditioning regimen included CY (200 mg/kg) and alemtuzumab (median dose, 60 mg) [n = 11], melphalan (MEL; 140 mg/m2) and alemtuzumab [n = 2], CY and rabbit anti-thymocyte globulin (rATG; 7.5 mg/kg) [n = 4], and CY alone (n = 1). Four deaths occurred early after transplant. There were three males and one female at median age at death of 51 years (range 24–68). The AHSCT-related deaths have been observed on days + 1, + 4, + 9, and + 15 after procedure. The causes of death included bilateral pneumonia followed by multi-organ failure in three patients and myocardial infarction in one. Three patients expired late during post-transplant follow-up, after 5, 21, and 42 months. The causes of death were disease progression in two patients and sudden heart attack in one. Eleven patients are alive after median follow-up after AHSCT of 42.0 months (range 0–95). Before proceeding to AHSCT in systemic sclerosis, there is a strong need to optimize patient selection to reduce toxicity. The administration of alemtuzumab should be avoided due to high risk of life-threatening infectious complications.”

Article on Heated Gloves for Hand Dysfunction in Scleroderma

This article was supplied to me courtesy of Dr. Allan Metzger*, Medical Director of RDL Reference Lab in Los Angeles.  It is an anecdotal guest column in the publication “The Rheumatologist” on the benefits of heated gloves on pain from Raynaud’s attacks, digital ulcers, and general hand discomfort from a patient with diffuse systemic scleroderma.… Continue Reading

Updated Version of “Suggested Protocol for a One-Year Trial of Therapeutic Plasma Exchange for Treating Systemic Sclerosis” Now Available

The purpose of this document is to provide background information for clinicians and patients who have reviewed the research literature on the use of therapeutic plasma exchange (TPE) to treat systemic sclerosis (SSc) and made the determination that this may be an appropriate treatment option.   Currently, the American Society for Apheresis Guidelines suggest that… Continue Reading

2017 EULAR Report Recommendations Summary Chart

I have had several requests for a simplified version of the new 2017 “Update of EULAR recommendations for the treatment of systemic sclerosis” paper that I mentioned in a post yesterday.  The paper includes a summary table that consolidates all of the new recommendations into a single table, sorted by symptoms.  You can view a slightly simplified… Continue Reading

Update of EULAR Recommendations for the Treatment of Systemic Sclerosis

Recently, the European League against Rheumatism (EULAR) released updated research-based recommendations for treating all aspects of systemic scleroderma.  It is freely available online here: http://ard.bmj.com/content/early/2017/04/25/annrheumdis-2016-209909?papetoc.  While this is a very technical article, most of it is actually pretty easy to understand as long as you are not intimated by the heavy use of abbreviations, universal in published medical research papers.… Continue Reading

“Role of Autoantibodies in Systemic Sclerosis: Relevance in Clinical Practice”

Thomas A. Medsger, Jr., MD University of Pittsburg School of Medicine I just found a recent presentation by Dr. Thomas Medsger on the clinical significance of autoantibodies in systemic scleroderma.  This is a slide show presented to rheumatologists in Arizona in 2014 and is the best summary of current research on this topic that I… Continue Reading

Prevalence and Incidence of Systemic Scleroderma in the US

It is very common to read in articles about systemic scleroderma that there are about 300,000 people in the US with this disease.  This number is completely wrong, according to numerous research studies.  The most complete data on the prevalence (number of diagnosed cases) and incidence (number of new cases per year) of systemic scleroderma comes from a… Continue Reading

Suggested Guidelines for Individual Trials of Therapeutic Plasma Exchange to Treat Systemic Scleroderma

Recently, two patients who are currently discussing therapeutic plasma exchange (TPE) with their physicians as a possible treatment option asked me for some information to give to their physicians that described the protocol that was used in the recently published case report (Harris et al. 2016) titled “Successful Long-Term (22 Year) Treatment of Limited Scleroderma… Continue Reading

“Diffuse”? “Limited”? “Overlap”? “Sine”?: Know Your Terms and Your Antibody Type

(This post is focused on systemic forms of scleroderma only. For a discussion on non-systemic forms, for example, linear scleroderma or morphea, or scleroderma-like disorders, see the Differential Diagnosis section of the Scleroderma FAQ.) 25 Years Ago The names used for referring to the scleroderma family of disease are very confusing for a number of… Continue Reading

Review: Therapeutic Plasma Exchange for the Treatment of Systemic Scleroderma

Last week I attended the 2016 annual meeting of the American Society for Apheresis (ASFA) in Palm Springs, CA and presented a research poster titled “Therapeutic Plasma Exchange for the Treatment of Systemic Scleroderma: A Comprehensive Review and Analysis”. The poster was well received, I was able to meet with vendors and others that are… Continue Reading