PasquiniM2 DM AID07vzsa

8/12/2019 PasquiniM2 DM AID07vzsa

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Marcelo C. Pasquini, MD,Marcelo C. Pasquini, MD, MScMSc

HVD05_1.ppt

Reporting Autoimmune DiseasesReporting Autoimmune Diseases

inin HematopoieticHematopoietic Stem CellStem Cell

TransplantationTransplantation

OutlineOutline

Review of autoimmune diseases

(AID).

Role of transplantation for AID

Data collection: points to consider.

AID when present as a comorbidity.

HVD05_2.ppt

IntroductionIntroduction

AID is a heterogenous group of diseasesthat have a link between the immunesystem and specific organ damage.

Spectrum of disease intensities great

impact on quality of life. Treatment:

Immunosuppressive agents:

MMF, CSA, tacrolimus, MTX, Cy

Immunomodulatory

Disease Modifying AntirheumaticDrugs (DMARDs)


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ClassificationClassification

Connective tissue diseases: Systemic Sclerosis (SSc) Systemic Lupus Erythematous (SLE) Rheumatoid Arthritis (RA) Juvenile Idiopathic Arthritis (JIA)

Neurologic Multiple Sclerosis (MS)

Hematologic Idiopathic Thrombocytopenic Purpura(ITP) Hemolytic Anemia (HA)

Gastrointestinal Inflammatory Bowel Diseases

Vasculitis Wegner Granulomatosis Poyarteritis nodosa

Endocrine Diabetes type I Thyroiditis

Multiple SclerosisMultiple Sclerosis

Neurologic AID

Direct myelin destruction and secondaryoligodendrocytes and axonal damage.

Symptoms start in early adulthood.

Variable disease course but most oftenprogressive

Estimate that 50% of patients with MSwill not able to walk within 15 years ofonset.

MS Clinical CourseMS Clinical Course


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MS ClinicalMS Clinical

EvaluationEvaluation

Diagnosis:Dissemination oftime and space

Performance ofactivities

MRI evaluation

CSF:

Oligoclonalbanding

Multiple SclerosisMultiple Sclerosis

Diagnosis Criteria (Poser)

Two attacks and clinical evidenceof two separate lesions or onelesion and other clinical sign ofthe disease

Symptoms:

Multifocal neurologic deficits

Treatment:

Interferon, antibodies,corticosteroids.

Systemic Sclerosis (Scleroderma)Systemic Sclerosis (Scleroderma)

Chronic multisystem disorder ofunknown etiology, characterized byskin thickening and several visceralorgans.

Target organs:

Skin, heart, lungs, GI, bloodvessels, kidneys.

Two subsets:

Diffuse cutaneous

Limited cutaneous


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SScSSc Disease EvaluationDisease Evaluation

Clinical features

Skin thickness

Raynauds

Sclerodactily

CREST

Disease markers:

ANA, Scl-70, ACA

Evaluation of organ involvement:

Echo, PFTs, creatinine clearance,

right heart cath, endoscopy

RaynaudRaynaudss PhenomenonPhenomenon


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Systemic LupusSystemic Lupus ErythematousErythematous

Collagen vascular disease with organ

damage by autoantibodies or immunecomplexes.

Women are affected in 90% of cases

Clinical Manifestation:

Fever

Skin rash

Muskoskeletal (myalgias and arthritis)

Kidney disease

Cardiopulmonary

Hematologic, Neurologic, GI, others

SLE EvaluationSLE Evaluation

Symptoms and signs

of organ involvement

Presence of autoantibodies or lowcomplement levels:

ANA, dsDNA, C3, C4

Rheumatoid Arthritis (RA)Rheumatoid Arthritis (RA)

Chronic multisystem disease withpersistent synovitis as the maincharacterisitic features

Women are 3 more affected than

men. Symmetric joint involvement

Other manifestations:

Rheumatoid nodules

Vasculitis

Pleural effusion and lungnodules

Neutropenia


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RA EvaluationRA Evaluation

Number of inflammed

joints Rheumatoid Factor

Radiologic evaluation

Transplantations for AIDTransplantations for AID

Objective:

Halt the autoimmunityprocess

Allogeneic

Graft versus autoimmunity

Patient population: severedisease and refractory to

previous therapies

Transplant IndicationsTransplant Indications

MS:

Except for primary progressive

Scleroderma:

SCOT trial and CTN 0602 SLE:

Reduced intensity auto (?)

RA:

Frequent post transplant relapses


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Most Common Diseases Reported toMost Common Diseases Reported to

the CIBMTRthe CIBMTR

713Other Autoimmune Diseases

-1Hemolytic Anemia

-2Evans Syndrome

2-Juvenile idiopathic arthritis

5-Rheumatoid Arthritis

4-Idiopathic ThromobocytopenicPurpura

142Systemic Lupus Erythematous

566Systemic Sclerosis74-Multiple Sclerosis

NN

AutologousAllogeneic

Da t a f r om No r t h a n d So u t h Ame r i c a n Cen t e r s

Points in Data CollectionPoints in Data Collection

Similar time points: Diagnosis, pre-mobilization, pre-

stem cell infusion, posttransplant follow up.

Diagnostic Criteria Prior Therapies Markers of disease activity

Laboratory and imagingevaluation

Evaluation of organ involvement: PFT, echocardiogram.

Disease specific scales

PrePre--TEDTED


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CIBMTR Disease InsertsCIBMTR Disease Inserts

Recipient Baseline data andPost-HSCT Data

MS

SSc

SLE

RA

JIA


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Scales and CriteriaScales and Criteria MS

Expanded Disability Severity Scale(EDSS)

Scripps neurological rating scale

Timed 25-foot walk

9-hole peg test

Paced Auditory Serial Addition Test(PASAT)

SSc

Modified Rodnan Skin Score

HVD05_3.ppt

EDSSEDSS

Scale from 0-10 according to thedegree of disability

O no disability

3 moderate disability

5 severe disability but ambulatory

7 unable to walk beyond 5 meters

9 bedridden with little interaction

10 death due to MS

Scales and CriteriaScales and Criteria

American College of Rheumatologydiagnostic criteria (RA, SSc and SLE)

RA:

EULAR Joint count

SLE:

SLE Disease Activity Index (SLEDAI)

Quality of life and functional Assessments

SF36

Health Assessment Questionnaire


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SLEDAISLEDAI

Difficulties in ReportingDifficulties in Reporting

Evaluations are less objectivethan evaluation of malignantdiseases.

Different diagnostic criteria

Several data points forcollection

Disease status evaluationrelies on different scales

AID as aAID as a ComorbidityComorbidity

Patients with minimally activedisease at time of

transplantation.

How the transplant affects thedisease.


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Case ReportsCase Reports

Diseases: RA, SLE, psoriasis,

myasthenia gravis, MS,ulcerative colitis, ITP,Sjogrens Sydrome

Most patient received anautologous HCT

The majority experience longlasting remissions.

Recipient Baseline DataRecipient Baseline Data

Reporting AID as a Coexisting diseasesReporting AID as a Coexisting diseases

What is relevant collect?

Patients with active disease

Patients requiring activeantirheumatic medications

Why collect?

Understand the effect of HCT in earlyAID

Understand whether the presence ofAID affects post transplantoutcomes.


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AID and Transplantation: summaryAID and Transplantation: summary

Primary indication Transplant is the main therapy.

Patients with more advanced AID

Coexisting

Presence as a comorbidity

Donor with autoimmune diseases

Adoptive transfer of autoimmunity

Post transplant chimerism and AID

ConclusionConclusion

HCT for AID is mainlyinvestigational.

Reporting data on AID is morelaborious and requiressubspecialties outside the transplantunit.

It is important to recognize AIDwhen present as a comorbidity.

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