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Bone Marrow Transplantationhttps://doi.org/10.1038/s41409-019-0430-7
PERSPECTIVE
General information for patients and carers consideringhaematopoietic stem cell transplantation (HSCT) for severeautoimmune diseases (ADs): A position statement from the EBMTAutoimmune Diseases Working Party (ADWP), the EBMT NursesGroup, the EBMT Patient, Family and Donor Committee and theJoint Accreditation Committee of ISCT and EBMT (JACIE)
AbstractOver the last 20 years, haematopoietic stem cell transplantation (HSCT) has been used to treat patients with severeautoimmune and inflammatory diseases whose response to standard treatment options has been limited, resulting in a poorlong-term prognosis in terms of survival or disability. The vast majority of patients have received autologous HSCT wherean increasing evidence-base supports its use in a wide range of autoimmune diseases, particularly relapsing remitting MS,systemic sclerosis and Crohn’s disease. Compared with standard treatments for autoimmune diseases, HSCT is associatedwith greater short-term risks, including a risk of treatment-related mortality and long-term complications. There is a need fora careful appraisal of potential benefits and risks by disease and transplant specialists working closely together with patientsand carers to determine individual suitability for HSCT. HSCT should be conducted in accredited transplant centres withrobust arrangements for long-term follow-up with both disease and transplant specialists. The aim of this open-accessposition statement is to provide plainly worded guidance for patients and non-specialist clinicians considering HSCT for anautoimmune disease, especially when treatment abroad is being considered. Recent technical publications in the field havebeen referenced to support the statement and provide more detail for clinicians advising patients.
Introduction
Autoimmune diseases (ADs) are a broad group of illnesseswhere the body’s immune system reacts against its owntissues and organs. The immune attack is followed bychronic inflammation and abnormal healing, which may beassociated with permanent organ damage, disability andpoor quality of life. In some cases, severe ADs may shortenlife expectancy or even be immediately life-threatening.
The types of organs affected vary between ADs. Forexample, systemic sclerosis, lupus, vasculitis and other
connective tissue diseases affect many organs, typicallycausing inflammation and scarring of the skin, heart,lungs, kidneys and other organs. Multiple sclerosis (MS)affects the brain and spinal cord, whereas Crohn’s diseaseaffects the gut.
Treatment with immunosuppressant drugs, includingdisease modifying treatments (DMTs), may be successful incontrolling the AD, but there is an increased susceptibilityto infection and organ damage, which add to the problemsof living with an AD.
Some patients have very aggressive forms of auto-immune disease which are poorly controlled by standardtherapies. In some of these severely affected patients, theremay be benefit in considering bone marrow transplantation(BMT), or, as it is now more commonly known, haemato-poietic stem cell transplantation (HSCT) [1–3].
HSCT is commonly used for the treatment of serious blooddiseases, including leukaemia, myeloma and lymphomas.Many haematology departments will provide HSCT as atreatment for these common indications, having carefullyweighed up the survival benefits and serious risks of HSCTagainst the non-transplant treatment options, such as che-motherapy and modern biological agents [4, 5].
The process of HSCT involves giving high doses ofchemotherapy and/or radiotherapy and then infusingmobilized blood, cord blood or marrow-derived (i.e.,’ hae-matopoietic’) stem cells from the patients themselves(autologous or auto-transplant) or from donors (allogeneicor allo-transplant) to rebuild the bone marrow and theimmune system, hopefully without the disease.
The HSCT process is summarised in Fig. 1. Recognisedearly complications include acute toxicities experiencedduring the procedure, such as infection, bleeding, anaemiaand organ toxicities. Blood transfusions are usually neededto support the patients during this phase of the treatment.Complications may be life-threatening and irreversible andtherefore HSCT should only be performed after a full dis-cussion of the risks and how they may be justified in termsof potential benefits from the treatment. Treatment-relatedmortality (TRM) risks depend on the type of transplant, thedisease being treated and the individual patient. Even inpatients treated in accredited transplant centres, TRM riskranges from around 1% in good risk patients undergoingautologous HSCT to considerably higher in patients withmore risk factors (e.g., severe organ damage or disability)and with allogeneic HSCT, where TRM risk commonlyexceeds 10%. Responsible treating clinicians need to openlydiscuss and individualise these risks for patients as part ofthe informed consent process.
The risks of the HSCT procedure are not limited to theinpatient phase prior to recovery of blood counts [6–8].Following hospital discharge after the HSCT procedure,given the risk of ongoing complications, especially the riskof sudden onset of serious and life-threatening complica-tions including infection, there should always be robustplans for follow up and urgent management of complica-tions. Patients should be able to self-refer into a haematol-ogy or other department familiar with HSCT on a 7-day,24-h basis, and the transplant unit should organise regularoutpatient monitoring in the first 2 years following theprocedure and thereafter if needed. The risks of infectionpersist for long periods after HSCT, and ‘prophylactic’(preventative) antibiotics are recommended for manymonths, or sometimes even life-long. Even though patientsmay feel well, some complications, including certain typesof pneumonia, may be rapid in their onset and progression
and much more challenging to treat when established.Patients need to be monitored, and provided with a con-sistent supply of prophylactic (preventative) antibiotics,which reduce hospitalisation for serious and sometimeslife-threatening complications. Vaccination should be con-sidered according to current guidelines for immunocom-promised patients [9].
Long-term monitoring is essential. The so-called ‘lateeffects’ of HSCT are common and may involve manyorgan systems, including glandular (endocrine), bone,cardiovascular, respiratory and neurological. HSCT canalso result in an increased risk of ‘new’ cancers, and havean impact on patients’ psychological and emotional well-being. Reproductive impairment can be frequent, andprogrammes for prevention of chemotherapy-associatedsterility should therefore be proposed prior to HSCT.Allogeneic HSCT may also be associated with risks ofgraft-versus-host disease, a potentially severe immunecomplication that may affect several organs. There is evena risk of new ADs (known as ‘secondary’ autoimmunediseases) after both autologous and allogeneic HSCT. Inrecognition of these ‘late effects’, the EBMT and otherorganisations have produced recommendations for long-term follow-up of patients who should have life-longfollow-up by clinicians familiar with ‘late effects’ fol-lowing HSCT, irrespective of where the procedure hasbeen performed [6–9].
Transplant centres should have approval of their ‘quality’by external accreditation organisations (Joint AccreditationCommittee of ISCT & EBMT, JACIE, or its North Amer-ican equivalent, the Foundation for Cellular Therapy,FACT), and there is published evidence supportingimproved safety and survival outcomes with accreditation[10, 11].
2. Stem cells collectedfrom peripheral blood
1. Mobilise stem cellsusing growth factor and
chemotherapy
7. Further follow-up asoutpatient for 2-3 monthswith regular blood tests
and medication
6. Support with bloodproducts and antibiotics(approximately 2 weeks)
5. Stem cells thawedand re-infused
3. Stem cells frozenuntil required
4. Conditioningchemotherapy tosuppress immune
system
Fig. 1 Summary of autologous HSCT procedure. In addition, robustlong-term follow-up arrangements should be individualised with eachpatient with both disease and transplant specialists
H. Jessop et al.
Haematopoietic stem cell transplantation(HSCT) for ADs
Over the last 20 years, HSCT has also been used to treatsevere ADs, as it is an effective means by which to suppressthe abnormal inflammation and associated scarring [1–3]. Itis also possible that HSCT can rebuild an altered immunesystem that does not regenerate the original AD, or at leastleads to a milder form and better disease control. To date,several thousand patients with ADs have been treatedworldwide with HSCT, with almost 3000 patients registeredin the EBMT registry (summarised in Table 1).
The main ADs treated are currently relapsing remittingMS, systemic sclerosis (SSc), systemic lupus erythematosus(SLE or ‘lupus’) and Crohn’s disease. Other rare immunedisorders have been treated in smaller numbers. The vastmajority of patients have been treated with autologousHSCT, with MS, SSc and Crohn’s disease being the maincurrent indications, reflected by the registrations for theyears following the publication of the EBMT ADWPguidelines and recommendations [12] in 2012 (Table 1). Asmall proportion of patients have received allogeneicHSCT, predominantly for rare diseases in the paedatricage group.
It should be emphasized that HSCT is not a form of‘regenerative’ stem cell therapy in the same way as manyscientists hope that stem cells may be used to rebuild neworgans. HSCT may lead to ‘re-setting‘ or ‘re-booting’ of theimmune system, which in turn may lead to improvement indamaged organs and tissues. HSCT works best if activeinflammation can be switched off, but the effect of HSCT maybe limited (or nothing at all) if there is no active inflammation.There is no proof that the blood stem cells can directly rebuildspecialised tissues, although in the absence of inflammationsome damaged tissues and organs may heal over time. Thepotential mechanism by which autologous HSCT leads to a re-setting or re-balancing of the immune system in autoimmunedisease is summarised in Fig. 2.
Although HSCT is common in many haematology andoncology departments, use of HSCT in ADs is relativelyrare and only a small number of transplant centres in eachcountry have a good level of experience in working withdisease specialists to select the most appropriate patientswith AD and then deliver the HSCT treatment as safely aspossible.
The type of HSCT almost always used in patients withsevere ADs is autologous HSCT (or ‘auto-transplant’).Auto-transplant is more safely delivered compared withallogeneic HSCT (or ‘allo-transplant’), which is a muchmore complex procedure requiring a donor. Even so, thereare significant risks and toxicities with autologous HSCT,which are an important consideration, especially if the ADhas already caused significant organ damage and disability.
In view of these toxicities and risks, it is very important thatpatients have a full discussion with their transplant andautoimmune disease specialist teams.
The individual patient’s response to HSCT is dependent onthe specific AD and the stage of the disease at the time oftreatment e.g., whether the inflammatory disease process canbe ‘switched off’ before it has become irreversible or hascaused permanent organ damage. Some patients have hadvery profound responses lasting many years, far more thanexpected from treatment with standard therapies, but othersrespond less well or experience a relapse of their AD soonafter the transplant. The benefit of HSCT in some ADs (MS,SSc and Crohn’s disease) has been shown in randomizedcontrolled trials (RCTs) against the best available conventionaltherapy for an AD. However, there is always need for moreevidence to help improve outcomes, and, when possible, ADpatients should be treated on ethically approved clinicalstudies.
Specific advice to patients considering HSCTfor their AD
We recommend that before starting treatment, patientsshould inform themselves firstly about current disease andtreatment guidelines, secondly about the professional com-petence of the clinicians and thirdly the experience andaccreditation status of the transplant centre.
The European Society of Blood & Marrow Transplan-tation (EBMT) has published a number of guidelines andrecommendations for the use of HSCT in ADs [11–13].Other organisations and individual health professionalshave also made recommendations in specific diseases[14–17]. Whereas these guidelines are primarily intendedfor a professional readership and therefore written in atechnical language, they are openly accessible to all.
Timing of HSCT is central to decision-making. Theseverity of the autoimmune disease, its treatment-resistanceand availability of other treatment options are essential con-siderations. However, HSCT needs to be considered beforeorgan damage caused by the autoimmune disease becomesirreversible (limiting benefits) and before the risks of HSCTare significantly increased by compromised vital organfunction. Balancing potential benefits and risks for individualpatients is often complex and requires disease specialists andtransplant clinicians to work closely together.
The EBMT recommend that patients ask their transplantand disease specialists to explain any aspect of the guide-lines, including whether treatment is in line with the pub-lished recommendations.
Treating clinicians should be active in the EBMT and/orother national and international professional societies,which means that they should follow published guidelines,
General information for patients and carers considering haematopoietic stem cell transplantation (HSCT). . .
report their outcomes to the EBMT (or equivalent profes-sional society) and maintain their professional competenciesin this highly specialized field. EBMT membership infor-mation is available from www.EBMT.org and accreditationstatus on www.JACIE.org websites.
Importantly, patients should ask about short- and long-term side-effects of the treatment and the level of risk ofdeath from the treatment and the severe AD to make abalanced decision. Patients should ask their transplant anddisease specialists about the experience of their transplant
Cryopreservation
Infections
Immune imbalance
Weeks –4 –2 –1 0 4 26 52
Immune resetting and rebalancingMobilization Conditioning
Inpatientrecovery
Outpati
ent m
onito
ring
Che
mot
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py
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ive
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rapy
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ion
Dietaryfactors
Autoimmunedisease
Patientselection and
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Self-reactive cellsattacking normal
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Otherenvironmental
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Fig. 2 Mechanism of immunere-setting with autologousHSCT in autoimmune diseases
Table 1 Summary of majorcategories and types ofautoimmune diseases treatedwith HSCT in the EBMTregistry, as per August 2018
Autologous HSCT for each categoryof autoimmune disease
Total adultpatients (1994–2018)
Total children (≤18 yearsat the time of transplant,1994–2018)
Total patients treatedin the last 6 years (allages, 2012–2017)
Autoimmune cytopenia, haemolyticanaemia, Evans syndrome,neutropenia and other
41 8 8
Type 1 diabetes mellitus 20 0 3
Miscellaneous other autoimmunediseases
18 3 9
The table summarises activity in autologous HSCT, which accounts for the vast majority of procedures.
Since 1996, 145 allogeneic transplants (from related and unrelated donors) have been performed forautoimmune diseases, approximately three quarters (73%) in children under the age of 18 for a range ofhaematological, rheumatological and other conditions
unit in general, whether the transplant unit is accredited (byJACIE, FACT or equivalent) and their specific experienceof treating your particular AD with HSCT. Ideally, thetransplant centres should have accreditation and have agood track record of experience and academic publication inthe disease for which HSCT is being considered.
Patients may also want to ask for an independent spe-cialist opinion or whether their case has had a documenteddiscussion with a wider group of professionals within amultidisciplinary team setting (i.e., an MDT meeting).Ideally patients should be seen and followed up in a com-bined clinic involving both disease and transplant specia-lists. Updates on specific ADs are provided on the EBMTwebsites listed at the end of this position statement.
One exception for being treated outside establishedguidelines is treatment on a clinical trial approved by aresearch ethics committee (REC) or institutional reviewboard (IRB), which may, for example, be testing whether anew technique might provide an advantage over existingtreatments. In this instance, patients will be counselled andasked to provide an informed consent for the trial.
Even if patients are not on a clinical trial, full informedconsent for treatment and also for data reporting is an EBMTrequirement for good clinical practice and data protection. Inorder for the transplant community to gain a better under-standing of the long-term side-effects of this treatment, it isimportant that transplant centres report data relating totransplant treatments to the EBMT or an equivalent registry.In the EBMT, patient data will be handled in an anonymous(and/or pseudonymised) fashion and in line with currentgeneral data protection regulation (GDPR).
Figure 3a–d summarises recent activity and JACIEaccreditation status in EBMT reporting centres for autologousHSCT procedures in ADs, both overall and for specific dis-ease categories. The information is primarily intended as aresource for clinicians in transplant centres and disease spe-cialists who wish to make contact with experienced EBMTcentres for advice, support, referral or other partnershiparrangements aimed at optimising patient care whilstaccommodating geographical considerations. The informationmay also be useful to patients and other carers; however,independent self-referral is strongly discouraged.
Treatment abroad
Patients should do their best to enquire whether treatment isavailable within their own health service, and, if not, theyshould question why. It may be that treatment is availableunder specific circumstances that do not cover specific typesof AD or a disease specialist may not consider it a bestoption. If treatment is planned outside of their home countrypatients should think carefully about their decision, as it
may not only have financial implications, but there may beexposure to unnecessary risks to their health and well-being.If treatment is unavailable in a patient’s home country, it ismore satisfactory if they travel and receive treatment withthe full support of a disease specialist and transplant spe-cialist who knows them well and is familiar with thecentre providing the treatment, or at least their generalpractitioner. This may enable direct communication withtransplant and disease specialist teams undertaking thetransplant. Independent self-referral by patients is stronglydiscouraged.
Even if the initial phase of the transplant is a success,patients should be aware that risks of treatment, such asinfection and organ damage, last for many months and, insome cases, years after the transplant procedure. As withHSCT in other settings, ongoing follow-up and support isrecommended, along with the need for rapid self-referral to alocally available transplant or other specialist haematologyunit in case of infections or other emergencies. The follow-upshould include the provision of monitoring for ‘late effects’ ofHSCT. These aspects of essential care may be compromisedif initial HSCT treatment is delivered outside of their homecountry, especially if communication between clinicians islimited. It is, therefore, important that patients specificallyaddress these issues prior to HSCT treatment as they mayrequire ongoing engagement with clinical services near theirhome (in both planned or unplanned settings, i.e., emergencyor urgent care).
Following return to home, some health services may notsupport monitoring and follow-up following HSCT,and this may lead to further financial commitment fromthe patient. The EBMT therefore recommend that beforeproceeding to transplants, patients carefully assessarrangements and support for medium- and long-termmonitoring and follow-up in their locally available healthservices.
Summary
● Haematopoietic stem cell transplantation (HSCT) hasbeen used in patients with severe autoimmune andinflammatory diseases whose response to standardtreatment options has been limited and associated witha poor long-term prognosis in terms of survival and/ordisability. The vast majority of patients have receivedautologous HSCT, with the use of allogeneic HSCTbeing rare and mainly in paediatric patients.
● Although a wide range of autoimmune diseases havebeen treated, the evidence base is greatest for the use ofautologous HSCT in relapsing remitting MS, diffusesystemic sclerosis and Crohn’s disease.
● HSCT may work by ‘re-setting‘ or ‘re-booting’ theimmune system, which in turn may lead to improvement
General information for patients and carers considering haematopoietic stem cell transplantation (HSCT). . .
120
Centre activity for AD overall, 2012-2017 (≥10 autologous HSCT procedures)
Centre activity, MS and other neurological AD, 2012-2017 (≥5 autologous HSCT procedures)
Centre activity for rheumatological AD, 2012-2017 (≥3 autologous HSCT procedures)
b
a
c
100
80
60A
uto-
HS
CT
40
20
0
120
100
80
60
Aut
o-H
SC
TA
uto-
HS
CT
40
20
0
30
20
10
0
MS CDOther AD
*JACIE accreditationSSc
SScOther rheumatological AD
MSMGCIDP
NMOOther neurol disorders
*JACIE accreditation
*JACIE accreditation
Katowice-CIC 677
Katowice-CIC 677
Katowice-CIC 677
Sydney-CIC 388
Singapore-CIC 434
Tricase~Lecce*-CIC 652
Linkoping-CIC 740
Moscow-CIC 411 (Ped)
Pamplona-CIC 737
Sydney-CIC 388
Sydney-CIC 388
Uppsala*-CIC 266
Uppsala*-CIC 266
London*-CIC 763
London*-CIC 763
London*-CIC 763
Goeteborg*-CIC 289
London*-CIC 205
London*-CIC 205G
enova*-CIC 217
Genova*-CIC 217
Barcelona*-CIC 214
Lund*-CIC 283
Lund*-CIC 283Heidelberg*-CIC 524
Heidelberg*-CIC 524
Heidelberg*-CIC 524Utrecht*-CIC 239
Wuerzburg*-CIC 712
Vilnius*-CIC 644
Firenze*-CIC 304
Oslo*-CIC 235
Toulouse-CIC 624
Basel*-CIC 202
Strasbourg-CIC 672
Tuebingen*-CIC 223
Tuebingen-CIC 223
Milano*-CIC 265
Milano*-CIC 813
Nijmegen*-CIC 237
Valencia*-CIC 663
Valencia*-CIC 282
Valencia*-CIC 663
Goeteborg*-CIC 289
Goeteborg*-CIC 289
Leuven*-CIC 209
Copenhagen*-CIC 206
Copenhagen*-CIC 206
Paris*-CIC 207
Bochum*-CIC 124
Bergen*-CIC 197
Hamburg*-CIC 614
Hamburg*-CIC 614
Palermo*-CIC 392
Palermo*-CIC 392
Torino*-CIC 305Toranto*-CIC 332
Torino*-CIC 305
Leiden*-CIC 203
Leiden*-CIC 203
Milan*-CIC 265
Nijmegen*-CIC 237
Paris*-CIC 207Bochum
*-CIC 124
Vilnius*-CIC 644
Vilnius*-CIC 644
Firenze*-CIC 304
Firenze*-CIC 304
Stockolm*-CIC 212
Stockolm*-CIC 212
Sheffield*-CIC 778
Sheffield*-CIC 778
Centre activity for AD overall, 2012-2017 (≥10 autologous HSCT procedures)
Centre activity, MS and other neurological AD, 2012-2017 (≥5 autologous HSCT procedures)
Centre activity for rheumatological AD, 2012-2017 (≥3 autologous HSCT procedures)
H. Jessop et al.
in damaged organs and tissues. HSCT works best ifactive inflammation can be switched off, but theeffect of HSCT may be limited (or no benefit at all) ifthere is no active inflammation. There is no proof thatthe blood stem cells can directly rebuild specialisedtissues, although the absence of inflammation mayenable some damaged tissues and organs to heal overtime.
● Compared with most standard treatments, HSCT isassociated with greater short-term risks, including a riskof treatment related mortality (TRM), and long-termcomplications (so called ‘late effects’) due to theintensity of the treatment. The risk of complications ishigher with autologous HSCT in some types of diseasesand in allogeneic HSCT. Risks increase with age, moreadvanced disease and the presence of other conditionswhich affect patients’ fitness. Decisions should beindividualised for each patient.
● Before decisions to proceed with HSCT can be made, itis essential to carry out a careful joint appraisal ofpotential benefits and risks by disease and transplantspecialists working closely together with patients andcarers. Alternative non-transplant treatments shouldalways be a consideration in decision-making.
● The EBMT and other professional societies havesummarised the evidence and produced guidelines andrecommendations as a resource to support centres andgood clinical practice.
● HSCT should be conducted in accredited units withrobust arrangements for early referral for urgent careafter the transplant and long-term follow up with bothdisease and transplant specialists. Centres shouldroutinely submit data on transplanted patients tointernational registries, such as EBMT.
● Treatment and follow-up on a clinical trial shouldalways be considered, if available within a reasonablegeographical reach of the patient’s home.
● Patients considering HSCT treatment abroad for theirAD are strongly advised to ensure that expectations inthis position statement are met by the centre offeringtreatment and that robust referral and follow-uparrangements are made with their local disease andtransplant specialists. Independent self-referral is stronglydiscouraged.
Acknowledgements We acknowledge the support of Eoin McGrath,JACIE Operations Manager.
20
d
CrohnCoeliac disease
15
10
5
0
Aut
o-H
SC
T
Milano*-CIC 265
Madrid*-CIC 615
Barcelona*-CIC 214
Leuven*-CIC 209Edinburgh*-CIC 228
London*-CIC 763
London*-CIC 768
Nottingham*-CIC 717
Amsterdam
*-CIC 588
Cordoba-CIC 238
Galway-CIC 408
Luebeck-CIC 367Nieuwegein-CIC 200
Prague-CIC 318
*JACIE accreditation
Centre activity for gastrointestinal AD, 2012-2017 (≥2 autologous HSCT procedures)
Centre activity for gastrointestinal AD, 2012-2017 (≥2 autologous HSCT procedures)
Fig. 3 a–d Activity and JACIE accreditation status in EBMT reportingcentres for autologous HSCT in autoimmune diseases. a Overall. bMSand neurological diseases. c Systemic sclerosis and rheumatologicaldiseases. d Crohn’s disease and gastrointestinal diseases. Activity isfor autologous procedures from 2012–2017 inclusive, i.e., reflectingpractice after the publication of EBMT ADWP Guidelines [12].Accreditation status is indicated by asterisks as active and/or with re-accreditation in progress as per December 2018 (www.JACIE.org).Centre identities according to CIC code are available on https://www.ebmt.org/ebmt-membership-list. The information is primarily intended
as a resource for clinicians in transplant centres and disease specialistswho wish to make contact with experienced EBMT centres for advice,support, referral or other partnership arrangements aimed at optimisingpatient care whilst accommodating geographical considerations. Theinformation may also be useful to patients and other carers; however,independent self-referral is strongly discouraged. MS multiple sclero-sis, SSc systemic sclerosis, CD Crohn’s disease, MGmyastheniagravis, CIDP chronic inflammatory demyelinating polyneuropathy,NMO neuromyelitis optica
General information for patients and carers considering haematopoietic stem cell transplantation (HSCT). . .
Conflict of interest JAS declares honoraria for speaking at educationalmeetings from Sanofi, Jazz, Janssen and Mallinckrodt. The remainingauthors declare that they have no conflict of interest.
Publisher’s note: Springer Nature remains neutral with regard tojurisdictional claims in published maps and institutional affiliations.
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The EBMT ADWP is dedicated to promoting clinicalactivities, teaching and performing translational research.Our focus is mainly on autologous/allogeneic HSCT,together with of novel forms of cellular therapy and newimmunosuppressive drugs in a specific challenge to inducetolerance, with the ultimate objective of better under-standing the possibility of resetting the immune responseduring treatment of severe autoimmune diseases. TheEBMT ADWP also has the strategic task of interacting withother autoimmune diseases (AD) specialists and theirrespective scientific societies.
The EBMT Nurses Grouphttps://www.ebmt.org/nurses-groupThe EBMT Nurses Group (NG) plays an essential role in
haematology and haematological stem cell transplantationnursing. The group was created 33 years ago and now hasover 750 members in more than 60 countries worldwidewith a principal nurse identified in almost each EBMTcentre. The EBMT NG’s mission is to enhance and valuethe nurses’ role all over the world, supporting and sharingknowledge through communication, advocacy, research,training and education. The group is dedicated to improvingthe care of patients receiving stem cell transplantation andworks towards promoting excellence in care throughrecognizing, building upon and providing evidence-basedpractice.
The EBMT patient, family and donor committeehttps://www.ebmt.org/patient-family-and-donor-
committeeThe objectives of the EBMT patient, family and donor
committee include supporting national patient advocacygroups, providing a pan-European platform for patientadvocacy groups including a patient dedicated link onEBMT website and providing advice on how to optimiseEBMT patient centred activities. In addition, the committeeaims to increase the numbers of international patients,family members and donors to access the EBMT patient,family and donor day at the annual meeting by promotingthe use of online webcasts and translation.
https://www.ebmt.org/accreditation/about-jaciehttps://www.ebmt.org/information-patients-donorsJACIE develops and maintain global standards for the
provision of quality medical and laboratory practice incellular therapy. Based on these standards, JACIE offersaccreditation to transplant programmes in order to encou-rage health institutions and facilities to establish andmaintain quality management systems impacting on allaspects of their activities and to engage in continuousimprovement. JACIE's primary aim is to promote high-quality patient care and laboratory performance in collec-tion, processing and transplantation centres through aninternationally recognised system of accreditation.
Paris EBMT OfficeMore technical information for clinicians, including
contact with key centres, is available from the Paris EBMTOffice via Manuela Badoglio, ADWP Study Coordinator,[email protected] who has access to haematolo-gists and disease specialists working in your region andcountries via the EBMT registry and can liaise with mem-bers of the EBMT.
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8 Department of Haematology, Institute of Haematology andOncology, IDIBAPS, Hospital Clinic, University of Barcelona,Josep Carreras Leukaemia Research Foundation, Barcelona, Spain
9 Department of Neurology, Sheffield Neuroscience BRC, SheffieldTeaching Hospitals NHS Foundation Trust, University of Sheffield,Sheffield S10 2JF, UK
10 Hematology and BMT Unit, San Raffaele Scientific Institute, ViaOlgettina 60, 20132 Milano, Italy