Peer-reviewed publications
Real‑world application of autologous hematopoietic stem cell transplantation in 507 patients with multiple sclerosis
9/24/21
Relapse free survival (RFS) at 5 years for RRMS and SPMS was 80.1% and 98.1%, respectively, while progression free survival (PFS) at 4 years for RRMS and SPMS was 95% versus 66%, respectively. For patients with RRMS, the EDSS signifcantly improved (p<0.0001) at each follow-up from a pre-HSCT mean of 3.87 to 2.51, 2.50, 2.41, 2.33, and 2.19 at 1, 2, 3, 4, and 5 years, respectively. For SPMS, the EDSS improved signifcantly only at 1 year but not thereafter. For SPMS, the mean baseline EDSS of 5.09 changed postHSCT to 4.85 (p=0.04), 4.88 (p=0.2), 4.92 (p=.27), 4.72 (p=0.07), and 4.2 (p=0.21) at 1, 2, 3, 4, 5 years, respectively...
Long-term Clinical Outcomes of Hematopoietic Stem Cell Transplantation in Multiple Sclerosis
2/23/21
To determine whether autologous hematopoietic stem cell transplantation (aHSCT) is able to induce durable disease remission in people with multiple sclerosis (MS), we analyzed the long-term outcomes after transplantation in a large cohort of patients with MS...
Hematopoietic stem cell transplantation found safe and effective for patients with Stiff Person Syndrome
1/21/21
Autologous non-myeloablative hematopoietic stem cell transplantation (HSCT) was safe and effective in a subset of patients with stiff person spectrum disorder (SPSD), according to a small open-label study, published in the December 14 online edition of Neurology...
Autologous haematopoietic stem cell
transplantation as a first-line disease-
modifying therapy in patients with
‘aggressive’ multiple sclerosis
12/11/20
Autologous haematopoietic stem cell transplantation (AHSCT) is an effective treatment for patients with multiple sclerosis (MS) who have highly active disease, despite the use of standard disease-modifying therapies (DMTs) ... AHSCT is safe and effective as a first-line DMT in inducing rapid and sustained remission in patients with ‘aggressive’ MS.
Autologous haematopoietic stem cell transplantation compared with alemtuzumab for relapsing–remitting multiple sclerosis: an observational study
9/6/20
The Kaplan-Meier estimates of the primary outcome measure ’no evidence of disease activity’ was 88% for AHSCT and 37% for ALZ at 3 years
Health economics and patient outcomes of hematopoietic stem cell transplantation versus disease-modifying therapies for relapsing remitting multiple sclerosis in the United States of America
7/15/20
Outside of the data, herein, no other studies on cost of HSCT for RRMS were found in the literature. HSCT mean total costs, based on our own hospital, were $85,184 (range $70,635 to $120,260). Mean revenue collected was $95,268 (range $16,544 to $173,204). In comparison, according to the literature, 2019 DMT costs in the USA ranged from $80,000 to $100,000 per year per patient. Compared to DMTs, studies of HSCT reported greater improvement in no evidence of disease activity, disability, and quality of life...
A pilot feasibility study of non-myeloablative allogeneic hematopoietic stem cell transplantation for refractory Crohn Disease
5/28/20
Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with severe anti-TNF refractory crohn disease: long-term follow-up.
Autologous nonmyeloablative hematopoietic stem cell transplantation for neuromyelitis optica
10/2/19
In the 11 patients whose baseline AQP4-IgG serostatus was positive, 9 patients became seronegative by the immunofluorescence or cell-binding assays available at the time; complement activating and cell-killing ability of patient serum was switched off in 6 of 7 patients with before and after HSCT testing. Two patients remained AQP4-IgG-seropositive (with persistent complement activating and cell-killing ability) and relapsed within 2 years of HSCT. No patient with seronegative conversion relapsed...
Man with severe autoimmune disease gets stem cell transplant at UW
6/5/19
Doctors diagnosed him with systemic scleroderma, an autoimmune disease that hardens skin and tissue of internal organs.
Psychiatric Care in Hematopoietic Stem Cell Transplantation
1/19/19
Patients with cancer frequently experience neuropsychiatric symptoms due to their medical illness or
its treatment. In recent decades, psychiatrists have become increasingly involved in the care of patients
with cancer. However, psychiatrists may be less familiar with hematopoietic stem cell transplantation
(HSCT), a distinct cancer treatment modality associated with multiple neuropsychiatric sequelae.
Stem cell transplant for severe scleroderma improves survival, quality of life
1/3/18
NIH-funded study finds transplantation superior to treatment with immune-suppressing drug.
Endocrinopathies after Allogeneic and Autologous Transplantation of Hematopoietic Stem Cells
4/30/14
Early and late endocrine disorders are among the most common complications in survivors after
hematopoietic allogeneic- (allo-) and autologous- (auto-) stem cell transplant (HSCT).
Lights and Shadows of Cyclophosphamide in the Treatment of Multiple Sclerosis
3/15/11
Cyclophosphamide (cy) is an alkylating agent used to treat malignancies and immune-mediated inflammatory nonmalignant processes...
HSCT PROTOCOLS FOR PEOPLE WITH MS
link to independent resource
HSCT Protocols for a variety of locations including A. A. Maximov aHSCT protocol for autoimmune diseases (Russia); Clínica Ruiz, The Mexican Method: aHSCT for MS patients (Mexico); Artemis Hospital aHSCT for MS patients (India); and Raffles Cancer Centre aHSCT for autoimmune diseases (Singapore); and MakatiMed aHSCT for MS patients (Philippines)
Autologous Peripheral Blood Stem Cell Transplant for Neurologic Autoimmune Diseases
(clinical trial in Colorado, US)
This phase II trial studies the side effects and how well carmustine, etoposide, cytarabine and melphalan together with antithymocyte globulin before a peripheral blood stem cell transplant works in treating patients with autoimmune neurologic disease that did not respond to previous therapy...
Outpatient Hematopoietic Grafting in Multiple Sclerosis Employing Autologous Peripheral Blood Stem Cells
(clinical trial in Mexico)
Multiple sclerosis (MS) is a chronic, inflammatory, debilitating disease that causes destruction of central nervous system (CNS) myelin...
New autoimmune diseases after autologous hematopoietic stem cell transplantation for multiple sclerosis
3/19/21
Secondary autoimmune diseases (2ndADs), most frequently autoimmune cytopenias (AICs), were first described after allogeneic hematopoietic stem cell transplantation (HSCT) undertaken for malignant and hematological indications, occurred at a prevalence of ~5–6.5%, and were attributed to allogeneic immune imbalances in the context of graft versus host disease, viral infections, and chronic immunosuppression...
The Cost Effectiveness of Immunoglobulin vs. Hematopoietic Stem Cell Transplantation for CIDP
3/22/21
Given the long-term treatment-free remission and better outcome measurements, autologous HSCT is more cost effective than long-term IVIG treatment in patients with chronic CIDP. However, costs will depend on patient selection, the HSCT regimen, and regional variations. Further analysis of the health economics, i.e., cost/outcome ratio, of HSCT as therapy for chronically IVIG dependent CIDP is warranted...
Autologous Hematopoietic Stem Cell Transplantation for Stiff-Person Spectrum Disorder
2/1/21
In this cohort, HSCT was safe, but the beneficial effect of HSCT was variable and confined predominately to participants with episodic spasms and normal tendon reflexes without simultaneous cocontraction of limb agonist/antagonist muscles who were not taking SSRI or SNRI antidepressants...
Rehabilitation Before and After Autologous Haematopoietic Stem Cell Transplantation (AHSCT) for Patients With Multiple Sclerosis (MS): Consensus Guidelines and Recommendations for Best Clinical Practice on Behalf of the Autoimmune Diseases Working Party, Nurses Group, and Patient Advocacy Committee of the European Society for Blood and Marrow Transplantation (EBMT)
12/11/20
Autologous haematopoietic stem cell transplantation (AHSCT) is increasingly used to treat people with multiple sclerosis (MS)...Although international guidelines and recommendations have been published for clinical and technical aspects of AHSCT in MS, there has been no detailed appraisal of the rehabilitation needed ... These expert consensus guidelines aim to address this unmet need by summarizing the evidence-base for AHSCT in MS and providing recommendations for current rehabilitation practice along with identifying areas for future research and development...
Cardiac safe hematopoietic stem cell transplantation for systemic sclerosis with poor cardiac function: a pilot safety study that decreases neutropenic interval to 5 days
7/1/20
We compared three fludarabine-based regimens for systemic sclerosis patients with a high-risk cardiac phenotype that according to EBMT criteria would be a contraindication for a high-dose cyclophosphamide (200 mg/kg) transplant regimen.
Hematopoietic stem cell transplantation for chronic infammatory
demyelinating polyradiculoneuropathy
6/18/20
Determine toxicity and efficacy of autologous hematopoietic stem cell transplantation (HSCT) for patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) who are dependent on intravenous immunoglobulins or plasmapheresis... A randomized trial is indicated to verify these results and confirm that HSCT reverses disability and offers longterm immune therapy independence...
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Lack of Efficacy of the Neutropenic Diet in Decreasing Infections among Cancer Patients A Systematic Review
12/12/19
Due to key advancements in chemotherapies,
immunotherapies, and transplant programs, there
have been stark advances in the survival of
cancer patients diagnosed with hematological
malignancies
Self‐reported changes in the expanded disability status scale score in patients with multiple sclerosis after autologous stem cell transplants: real‐world data from a single center
8/8/19
In order to reset the immune system to baseline function, autologous hematopoietic stem cell transplantation (HSCT) has been performed in patients with multiple sclerosis...
Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis
1/15/19
Conclusions and Relevance In this preliminary study of patients with relapsing-remitting MS, nonmyeloablative HSCT, compared with DMT...
Things We Do For No Reason- Neutropenic Diet
3/15/18
The “Things We Do for No Reason” series reviews practices
which have become common parts of hospital care but which may provide little value to our patients.
Stem Cell Transplantation for Refractory Crohn Disease
6/21/16
Letter to the Editor published by JAMA
Neutropenic diets in hematopoietic stem cell transplantation
3/21/12
Hematopoietic stem cell transplantation (HSCT) is an aggressive therapeutic procedure that consists in
the administration of high-dose chemotherapy (conditioning regimen) sometimes associated with
radiotherapy prior to an infusion of hematopoietic stem cell precursors.
Psychological Consequences of Hematopoietic Stem Cell Transplant
5/25/09
To address the psychological impact of the transplant on quality of life including physical,
psychological, social and spiritual for the patient and caregiver and to discuss the nurse’s emotional
labor of caring and compassion fatigue for such an intense vulnerable population.
Immunotherapy and Autoimmune Diseases
link to Northwestern Medicine
The Immunotherapy and Autoimmune Diseases Program is committed to finding the answers to today's most disabling and puzzling medical problems.
Best Available Therapy Versus Autologous Hematopoetic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) (BEAT-MS)
(clinical trial at 20 locations in the US)
This is a multi-center prospective rater-masked (blinded) randomized controlled trial of 156 participants, comparing the treatment strategy of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) to the treatment strategy of Best Available Therapy (BAT) for treatment-resistant relapsing multiple sclerosis (MS). Participants will be randomized at a 1 to 1 (1:1) ratio. All participants will be followed for 72 months after randomization (Day 0, Visit 0).