I was put in contact with someone with MS who was about to spend ~£60,000 by going abroad for HSCT. She lives in one of the home counties, i.e. a county that abuts on the outer perimeter of London, and hence could be referred into one of the London MS Centres for an opinion. In summary, she is on natalizumab and had a relapse. She was told by her consultant that as her MRI had no enhancing lesions she was not eligible to be referred for HSCT. This is incorrect the London MS-AHSCT Collaborative Group’s current eligibility criteria for AHSCT (autologous haematopoietic stem cell therapy) state either the presence of Gd-enhancing lesions or an increase in T2-lesion load.
Please note that the London MS-AHSCT stress that they currently view this as an exceptional therapy for some people with MS, rather than a standard treatment; and that neither NICE nor NHS England have ‘green lighted’ this therapy for routine use in any form of MS. However, MS is green-lighted as one of the autoimmune diseases that could be treated by haematologists under their own NICE guidance. However, there is a current NICE health technology appraisal proposing this at present.
If you are interested in this treatment it is important that you consult with your Neurology Team in the first instance.
Patient Eligibility Criteria Adopted by the London MS-AHSCT Collaborative Group
The eligibility criteria are overall aimed at selecting patients who have failed approved treatments of high efficacy or have none available to them and have recently presented evidence of inflammatory CNS disease activity; and who could undergo AHSCT with an acceptable estimated level of risk of adverse events. Justification for each of criteria is supported by evidence from AHSCT trials and observational studies.
I. Diagnosis of MS made by a neurologist
II. Able to walk, needing at most bilateral assistance to walk 20m without resting
III. In relapsing MS (RMS), failed one licensed disease modifying drug of high efficacy (currently including alemtuzumab and natalizumab) because of demonstrated lack of efficacy
IV. New MRI activity within last 12 months
2. Disease duration ≤15 years from diagnosis of MS
3. Diagnosis of MS according to McDonald’s criteria
4. For PPMS, CSF OCB+
5. For RMS, failed at least one licensed disease modifying drug of high efficacy (‘Category 2’ as defined by Scolding N, Barnes D, Cader S, et al. Pract Neurol 2015;15:273–279; currently including alemtuzumab and natalizumab) because of demonstrated lack of efficacy (as evident from relapse, MRI activity as defined below at Point 7, or EDSS increase) after being on DMT for at least 6 months
6. EDSS score 0-6.5
7. Inflammatory active MS as defined by ≥1 Gd+ (>3mm) lesion (off steroids for one month) or ≥2 new T2 lesions in MRI within last 12 months
8. Approved by the MDT
a. Eligible for an ethically approved clinical trial where AHSCT is offered as one of the treatment arms
b. Unable to adequately understand risk and benefits of AHSCT and give written informed consent
c. Prior treatment with total lymphoid irradiation and autologous or allogeneic hematopoietic stem cell transplantation London MS-AHSCT Collaborative Group – Patient Eligibility Criteria Final V.3. – 8/12/2015
d. Contraindication to MRI including but not limited to metal implants or fragments, history of claustrophobia or the inability of the subject to lie still on their back
e. Poorly controlled depression or recent suicidal attempt
f. Presence of any active or chronic infection
g. Unable to walk 20mt with or without support, or wheelchair dependent
h. Any significant organ dysfunction or co-morbidity that the Investigators consider would put the subject at unacceptable risk
9 thoughts on “#CaseStudy: Are you eligible for HSCT in London?”
Great post No COIIf Barts blog defends agressive treatment early in disease course,so called IRT,theraphy ,(Alemtuzumab ,Cladribine,etc)Why dont you consider Hsct a first line theraphy ?"Invert pyramid" aproachThanks Luis
At a personal level I don't have a problem with HSCT first-line now that it has become much safer. I suspect the new safety data may make it as safe, or safer, than alemtuzumab and more cost effective. However, we need controlled data to convince the the community and our peers.As a first step we should be grateful to have the ability to prescribe it to our patients who fulfil the treatment criteria, albeit quite restricted at present.
Thanks for replying Dr G Also would like your comment onLipoic acid in secondary progressive MShttp://nn.neurology.org/content/4/5/e374.short?rss=1Thanks Luis
"At a personal level I don't have a problem with HSCT first-line now that it has become much safer.".Utter weak nonsense! The safety of the treatment has remained near constant. What an underhand way to skirt around your early hypocritical stance.
Will the BMT units accept out of area patients?
Yes, as far as I am aware they will clear funding with the local clinical commissioning group (CCG) to fund the procedure. I suspect there is always a risk the CCG may say no.
Welsh and Scottish CCG's have said no to all HSCT referrals to date. As far as I know, every patient from England who has been accepted for HSCT in London, has not had any funding difficulties.If you meet the criteria, but live in Scotland or Wales, I would seriously suggest moving to England before applying.
Re: "Welsh and Scottish CCG's have said no to all HSCT referrals to date."May be they haven't realised yet that HSCT is cheaper than current DMTs.
I'm surprised to see that OCB+ PPMS patients are included as eligible patients. Is this based on data indicating efficacy of HSCT for PPMS patients?Almost all of the studies I've read have indicated that HSCT has little impact on disease progression for patients with strictly progressive disease. The Swedish longitudinal studies, among others, show much higher efficacy in patients with active RRMS or SPMS with enhancing lesions, but little efficacy for patients with PPMS.I would love to see any hard data indicating that HSCT would be of help to PPMS patients. Does any exist that I am not aware of? I know there are numerous PPMS patients online who have had the treatment and say they've seen positive outcomes, but anecdotal evidence can't be taken as gospel, as was recently evidenced by the failure of MIS 416 after years of glowing patient testimonials.Of course, I would love to find out that HSCT is effective in patients with PPMS, so any info provided will be tremendously welcome.