Download Ed Harris – My Story - Scleroderma Education Project

Ed Harris – My Story
Updated: June 21, 2016
[email protected]
My Medical History
Diagnosis and Early Symptoms
IwasdiagnosedwithCRESTsyndrome(oldnameforlimitedsystemicscleroderma)inJanuaryof
1990,buthadinitialsymptomsforaboutfiveyearsbeforemydiagnosis.Myapproachfromthe
onsethasbeentoreadeverythingIcouldfindonthediseaseanddecideonmyowncourseof
treatment.(IhadtochangephysiciansbeforeIcouldfindonewhowaswillingtoletmetake
chargeofmyowntreatments!)
Theconventionalmedicalapproachfortreatingthisdiseaseistowaituntilsymptomsdevelopand
thentrytotreatthesymptoms.Thismadenosensetomeatthetimeofdiagnosisandstilldoesnot.
EvenbeforeIunderstoodwhatInowdoaboutthedisease,itwasobvioustomethattryingto
preventthediseasefromprogressingwouldpotentiallybe“easier”thandealingwithsymptoms
aftertheyarose.
In1991,Ibegantohavesevereheartburn,whichischaracteristicofCRESTsyndrome.Thisis
becausetheloweresophagealsphincterlosesmuscletoneaspartofthedisease,resultinginacid
reflux,i.e.,thebackupofstomachacidintotheesophagus,whichiswhatheartburnis.AtthetimeI
wastakingProcardia(nifedipine)totreatmyRaynaud’ssymptoms.Myphysicianhadeithernot
knownorfailedtomentiontomethatProcardiaandallothersimilarmedicationshaveasideeffect
ofreducingtheloweresophagealsphincterpressure,thusincreasingheartburn!Idroppedthe
Procardia,whichimprovedtheheartburn,butmyRaynaud’sgotworse.So,backtotheresearch
literature...Iquicklydiscoveredthatsomeresearcherswereexperimentingwithadrugcalled
YohimbinefortreatingRaynaud’s(thedrughasbeenusedforcenturiesasanaturalaphrodisiac
andwasthenmostlyusedtotreatcertainformsofimpotence.).ItturnsoutthatYohimbinedoes
nothavethesideeffectproblemthatProcardiadoes,andin1992,Ibegantouseitinsteadof
nifedipinewithalmostasgoodaresultsforhelpingmyRaynaud’ssymptoms.Meanwhile,my
heartburncontinuedtogetworseandwasnotfullycontrolledbylargedosesofPrilosec
(omeprazole).Also,bylate1992Ihadbecomechronicallychilled,tothepointwhereIwaswearing
sweatersinthesummerandraisingtheheatinmycarsomuchmyfamilywasconstantly
complainingaboutsuffocatingfromtheheat!
Search for a Potential Treatment
Bylate1992,Iwasstartingtofeelincreasinglyhopelessasmysymptomscontinuedtoprogress.
MywifeandIwenttoseethemovie“Lorenzo’sOil”andIleftthemovieinspiredtotrytofinda
treatmentapproachthatperhapsmyphysiciansdidn’tknowabout.So,inearly1993,Ibegana
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muchmorethoroughliteraturesearchtoseeifIcouldgetabetterunderstandingofthedisease
processandperhapsdiscoverawaytoeitherstopordelaythenormaldiseaseprogression.
However,youhavetothinkabouthowchallengingthiswasin1993.TherewasnoInternetand
whileIhadaccesstoamedicalschoollibrarywhereIlived,doingaliteraturesearchmeantstarting
withcardcatalogsandultimatelygoingthroughmanyboundvolumesofmultiplerheumatology
journalsasafirststep.ThistaskwasveryslowanddifficultandIwasincreasinglyfeelingthatit
wasaninsurmountabletask.
Then,throughanamazing“payitforward”story,everythingchanged.IwasthefounderandCEOof
afairlylargesoftwarecompanybackthenandwasonanairplanetriptoatradeshowthatwewere
exhibitingat.Idon’trecallhowthiscameupbutIendeduptalkingwiththepersonsittingnextto
meaboutmymedicalsituationandwhatIwastryingtoaccomplishinmyreviewofthemedical
literature.ItturnsoutthatthisgentlemanwasalsotheCEOofasoftwarecompany,butinhiscase,
hiscompany(SilverPlatter)soldMedline–themedicalsearchcatalog–tomedicallibrariesand
researchfacilitiesonCDRomforseveralthousanddollars.Hesaid“tellyouwhat,whenIgetback
totheofficenextweek,IwillsendyouafreecopyofMedlinesoyoucanbetterdotheresearchyou
aretryingtodo”.HefollowedupandabouttwoweekslaterIreceivedhisincrediblygenerousgift,
whichliterallychanged(andprobablysaved)mylife!(Asasidenote,in2014,Itrackedhimdown
withgreatdifficultyandtoldhimeverythingthathastranspiredsincebecauseofhisgenerousgift.
Hetoldmelaterthathewasreallymovedafterreadingmyemail.)
Armedwithavastlyimprovedwaytodomedicalresearch,Ilocatedsomelittleknownresearch
studiesfromtheNetherlandsthatshowedaverysignificantfindingaboutsclerodermathatappears
tohavebeenmissedbyvirtuallyallsclerodermaresearchersatthattime.Itturnsoutthatin
sclerodermapatientswithRaynaud’s,theseresearchershaddiscoveredthattheredbloodcells
clumptogetherabnormallyascomparedtoeithernormalpeopleorevenpeoplewithprimary
Raynaud’s(samesymptoms,butnounderlyingseriousdisease).Theywereusingatreatment
calledplasmapheresis(alsocalledtherapeuticplasmaexchangeorTPE)totreattheRaynaud’s
symptoms,andhaddiscoveredthatithadsignificantlyreducedsclerodermarelatedRaynaud’sbut
noeffectonprimaryRaynaud’s.Moreimportantly,theTPEtreatmentsresultedinareturnto
normalredbloodcellaggregationthatlastedforasignificantamountoftime(3to9months)
followingasingleseriesofjustfourweeklyTPEtreatments.Icalledtheresearchersaswellasa
clinicianintheNetherlandsandlearnedthattheyhadbeenusingitclinicallythereforawhilewith
goodsuccesswithCRESTpatients,butnotasgoodresultswithdiffusesclerodermapatients.With
diffusepatients,thistreatmentapproachdidappeartoworkwellinitiallyevenforthemoresevere
patients,butonlyiftheTPEtreatmentsweredoneonaweeklybasis.Unfortunately,aweekly
treatmentfrequencycannotbesustainedoverthelongtermwithoutsignificantproblemsfrom
suppressionoftheimmunesystem.(Thissameproblemisseenincurrenttreatmentapproachesto
sclerodermathatdependmostlyontheuseofimmunosuppressantdrugs.)Incontrast,thereduced
frequencyoftreatmentsappearedtoworkwellinpatientswithlimitedsclerodermawithoutanyof
theimmunosuppressionproblemsseenwithlong-termweeklyTPEtreatments.
Thisgavemeaclueastowhatmightbegoingoninscleroderma.Bydoingsignificantlymore
literatureresearch,Iwasabletoconvincemyselfthatmost,ifnotall,sclerodermasymptomscould
beaccountedforsimplybythepotentiallong-termeffectsofabnormalredbloodcellaggregation.
Ironically,atthetimeIdidthis,theideaofprogressivevasculardamageasatriggerforthe
developmentofsclerodermasymptomswasnotwidelydiscussedintheresearchliterature,butin
thepastfewyears,Ihavenoticedthatthisisnowthegenerallyacceptedtheoryastothe
mechanismofdamageinsystemicscleroderma.However,Ihavetothisdatestillnotseenany
mentionoftheabnormalredbloodcellaggregationasapotential“firstcause”.
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Plasmapheresis,bytheway,isasimplebuteffectiveprocedure.Whatisdoneistoinsertone
needleintoaveinononearm,andanotherneedleintoaveinontheotherarm.Acontinuousflow
machineremovesbloodfromonearm,separatesouttheredandwhitebloodcellsandplatelets
fromtheplasmausingacentrifugalseparationmechanism,discardstheplasma,re-mixesthe
red/whitebloodcells/plateletswitheithersterilizedalbuminordonatedplasma,andputsthenew
mixturebackintheotherarm.Ittakesabouttwohours,ismildlyuncomfortableoncetheygetthe
needlesin,andleavesmefeelingtiredforafewhoursaftertreatment.Theusualrationaleforusing
plasmapheresistotreatandautoimmunediseaseisthatittemporarilyreducescirculating
antibodiesandotherpossiblesubstancesthatmighthavearoleinthedevelopmentofdisease
symptoms.However,whilethismaybeafactorintheimprovementsseenfromusingTPEwith
sclerodermapatients,Inowbelievethattheresearchsupportsadifferentmechanismofactionthat
mayaccountformostoftheseimprovements.
Iputtogetheratreatmentplanthatincludedannotatedresearchcitations,submittedittomy
physicians,andaftermonthsofeffortactuallymanagedtoconvincemyinsurancecompanytofund
aone-yeartrialofthistreatmentapproach.Itisworthnotingthatwhilemyteamofphysicians
agreedtotrythissincetheyhadnothingelsetoofferthattheyfeltwouldbeeffectiveinslowing
downdiseaseprogression,theydidnotexpectittohaveanybeneficialeffects.
IbegantreatmentsinNovember1993.Basedontheresearchresultsfromtheoriginalstudiesand
adiscussionwiththeclinicianstryingthisintheNetherlands,Idecidedonaninitialtreatmentcycle
thatconsistedofonetreatmentperweekforfourweeks,followedbyatwo-monthlayoff,thenthe
cyclerepeats,foratotalof16treatmentsduringthisfirstyear.
Initial Results
AfteroneyearoftreatmentsmyrefluxwassignificantlyimprovedandwhileIwasstillsomewhat
chronicallychilleditwasnotasbadasithadbeenbeforestartingthetreatments.Aftertwoyears
oftreatmentsmyrefluxwasundercompletecontrolwithaverylowdoseofPrilosec.And,Iwasno
longerchronicallycoldatall.Theseareniceresults,buttheyaresubjective.Ofmuchmore
significanceisthefactthatmyhematocritvalue(aquantitativemeasureofthepercentageofred
bloodcells),whichhadbeenabnormallylowfortheprevious8years,returnedtoanormalrange
followingthefirstroundoffourplasmapheresistreatments.Thiswascompletelyunexpectedsince
normallywithplasmapheresistreatmentsyoumayseeaslightdropinhematocritlevelsbecauseof
minorbloodlossfromtheprocedure.WhenIfirstsawthissuddenincreaseofhematocritafterthe
firstroundoftreatments,Ispeculatedthatthereasonforthismightbebecausetheautomated
equipmentusedfordoingbloodcountsmightbecountingsmallclumpsofredbloodcellsasa
singlelargeredbloodcellandthatoncetheclumpingwasbrokenup,theredbloodcellcount
wouldbemoreaccurate.Iwasfinallyabletoconfirmin2014thatthisisexactlywhathappenswith
clumpedredbloodcellsbytalkingwithanengineeratthecompanythatmakesthecellcounting
equipmentthatwasinusebackin1993(Beckman-Coulter).
Oneothersignificantquantitativemeasurethatsupportedthetheorythattheplasmapheresis
treatmentswerestoppingtheprogressionofthediseaseistheresultsofrepeatedpulmonary
functiontests(PFT).InJune1994,IhadaninitialPFTthatshowedthatwhilemostmeasures,e.g.,
lungcapacity,werenormalorabovenormal(Iexercisealot),thegasexchangeabilityofmylungs
haddeclinedtoabout68%oftheexpectedvalue.Thisiscommonwithlimitedsclerodermaandifit
continuestoreduceovertime,thiscanbeveryserious,ultimatelyleadingtopulmonary
hypertension,whichisevennowverydifficulttotreat.Aboutoneyearlater,inMay1995,I
repeatedthePFT.Theresultsofthiscriticalmeasurewereunchanged.Atthattime,Iconsidered
thistobethebestpossibleresultsincethesechangestothelungsarenormallyirreversibleand
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progressive.InAugust1997IrepeatedthePFTagain.Again,allothermeasuresexceptgas
exchangewerenormalorabovenormal.However,thistimemygasexchangemeasurehad
increasedsignificantly,uptoabout76%ofexpected.Thiswasanunexpectedresultandcertainly
verygoodnews.Itsuggestedthatmylungswereslowlyhealing.MymostrecentPFTwasdonein
December2000.Thistimemygasexchangemeasurehadincreasedto81%oftheexpectedvalue,
whichisinthenormalrangeof80%to120%.
Evidence for Effectiveness of Treatments - Part 1
In1997Iwasforcedtostopmyplasmapheresistreatmentsagainstmywishes.Whilemyinternist
andrheumatologistwerethenbecomingincreasinglyconvincedthatthetreatmentswereeffective
atstoppingoratleastdelayingtheprogressionofmylimitedscleroderma,oneofthephysicians
whowasinchargeofthetransfusionservicesunitwheremytreatmentswerebeingdonehad
alwaysbeenhostiletothetreatments,consideringthemawasteoftimeandthatallofmyso-called
improvementswereplaceboeffect.Heconvincedmyphysicianstostopthetreatmentstoseewhat
wouldhappen.Theresultswereverysignificant.Aboutsixmonthsaftermytreatmentswere
suspended,myheartburnsymptomsstartedtoreturnforthefirsttimeinyears.Thisgavemymain
physiciansenough“ammunition”tojustifyputtingmebackonthetreatments.Asbefore,ittook
aboutoneyearoftreatmentsfortherefluxtogetunderfullcontrolagain.Whilethisforced
vacationfromthetreatmentswasnottomyliking,itactuallyhadamajorimpactonhowmy
physiciansviewedtheeffectivenessofthetreatments.
[Technicaldigression…Inadditiontobeingacomputerexpert,Iamtrainedasaresearchclinical
psychologist.Duringmyresearchtrainingwelearnedhowtocorrectlydesignresearch
experiments.Obviouslyasinglecasestudyisneveranymorethansuggestivethatatreatmentmay
beeffectiveinthegeneralpopulation.However,thereisasinglesubjectresearchdesignthatcan
beusedtoestablishthatanyeffectsseenafteratreatmentisinitiatedislikelytobefromthe
treatmentandnotjustcoincidence.Basically,ifyoutakeabaselinemeasureofacondition,adda
treatmentandgetachangeinthatmeasure,thenremovethetreatmentandseeareturntothe
baselinelevel,andthenre-introducethetreatmentandgetthesamechangeinthemeasure,thereis
averysignificantlikelihoodthatthetreatmentcausedthechange.ThisiscalledanABABReversal
designandiswellestablishedasavalidresearchtechniqueinsingle-subjectresearchdesign.And
thisisexactlywhathappenedwhenmytreatmentswereremovedandthenreintroduced.]
Evidence for Effectiveness of Treatments - Part 2
In2003,thingswerecontinuingtogoextremelywell.Ihadnoresidualsymptomsotherthanvery
mildRaynaud'sandslightrefluxcompletelycontrolledbylowdosesofPrilosec(seenotebelow).
SoIdecidedtoseeifIcouldcutbackthefrequencyoftreatmentsabit.Ihadhistoricallyfolloweda
treatmentcycleofoneonetreatmentperweekforfourweeks,followedbyalayoffalternating
between8and9weeks,soastoaverageatwo-monthgapbetweentreatmentrounds.Idecidedto
graduallyincreasetheintervalbetweentreatmentrounds.Istartedbydoingtwotreatmentrounds
witha9-weekgapwithnoproblems.Thiswasfollowedbytwotreatmentroundswitha10-week
gap.ThisalsoseemedtogowellsoIincreasedthegapagain,to11weeks.However,thistimemy
heartburnsymptomsbegantore-occuragain.Iimmediatelywentbacktotheoriginaltreatment
cycle,andaftertwotreatmentcycleswithan8-weekgap,myheartburnsymptomsagain
disappeared.SincethenIhavestayedattheoriginaltwo-monthtreatmentgapwithnoother
problems.
Onimportantsidenoteonprotonpumpinhibitors(PPIs)suchasPrilosec(omeprazole):althoughI
hadnoheartburnsymptomswithfairlylowdosesofPrilosec,wheneverIstoppedthemedicineI
startedtohaveheartburnsymptomsagain.Iassumedthattherewasstillsomeresidualweakening
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oftheloweresophagealsphincterevenwiththecontinuedplasmapheresistreatments.However,it
turnsoutthatwasnotthecaseatall.Mywife,whoisaphysician,developedanulcerandhadtogo
onPrilosecaswell.AsitisnowknownthatmostcasesofulcersarecausedbyanH.Pyloriinfection
andthatappropriateantibiotictreatmentscancompletelycuretheunderlyingcauseoftheulcers,
shereceivedacourseoftreatmentsthatcompletelycuredherulcers.However,shewasalso
unabletostopPrilosecwithoutherheartburnsymptomsreturning!Shedidsomeresearchand
discoveredthatmedicinessuchasPrilosecactuallycreatetheirownneed!Apparently,ifyouput
healthyvolunteerswithnoheartburnsymptomsonPrilosecandthenstopit,theparticipantsinthe
studyactuallydevelopreboundheartburnsymptoms!Armedwiththisknowledge,weboth
decidedtotaperoffPrilosecVERYslowlyoverathree-monthperiodbackin2006.Thisworked
andneitherofushaveeverneededtogobackonPrilosecagain.Iamcompletelyfreeofany
heartburnproblemswithnomedications.ItisverylikelythatIcouldhavestoppedthePrilosec
backin1996hadIknownthis.
The Present Day…
AsIeditthisarticle(February2016),Iamnow68andinexcellenthealthwiththeonlyremaining
scleroderma-relatedsymptombeingverymildRaynaud’s(whichissayingsomethingbecauseIlive
inMadison,WIanddealwithverycoldwinters)!Accordingtomyinternist,Iamthehealthiest
patientinmyagebracketinhisentirepractice,inspiteofthefactthatIstillhaveaformaldiagnosis
oflimitedsystemicscleroderma.Iamveryactive(Iplaytenniseverydayandalsoworkoutonmy
ellipticaleverydayaswell).Medicarecontinuestopayformymedicaltreatmentswithoutany
problemsandthecurrentplanistostayonatreatmentfrequencyof16treatmentsperyear
indefinitely,oruntilamoreeffectivetreatmentbecomesavailable.Todate,Ihavehadmorethan
350plasmapheresistreatmentswithveryfewissuesorproblems.Theonlyexceptionwasone
incidentthatarosefromdefectivetubingthatleadtosomebloodlossthatwaseasilydealtwith.
ThisisnowaroutineprocedurethatIgothrough16timesayear.Iconsiderthisaverysmallprice
topayforbeinginexcellenthealthmorethan30yearsafterfirstdevelopingsymptomsoflimited
systemicscleroderma.
The Rest of My Story
The Scleroderma Education Project
In1995,astheInternetwasinitsinfancy,Isearchedanddiscoveredthattherewasessentiallyno
informationavailableonlineforsclerodermapatientsthatcouldbereadilyunderstoodbysomeone
withoutamedicalbackground.IwasabletoobtainanupdatetotheMedlineCDROMlibrarythatI
hadoriginallyreceivedin1993,reviewedallnewscleroderma-relatedstudiessince1993,and
wroteandpublishedonlinethefirsteditionofapatienteducationdocumentonsclerodermatitled
theSclerodermaFAQ.ItwontwopatienteducationawardsandwastranslatedintoSpanishover
thenextfewyears.ThelastmajorupdatetotheSclerodermaFAQwasdonein2009sincegood
patienteducationinformationwasfinallybecomingavailablefromanumberofsourcesincluding
theSclerodermaFoundation,theMayoClinic,andJohnsHopkinsUniversity.
InOctober2013,IdecidedtodoanupdatetotheSclerodermaFAQasaprequeltoupdatingthe
Wikipediaarticleonscleroderma,whichisnotverygood.Afterreviewingthemajorscleroderma
educationalwebsites,Irealizedthattherewasahugeamountofimportantinformationthatwas
notbeingincludedonanyexistingwebsite.Alotofthisinformationwasfairlytechnicalbutalso
importantforpeoplethatwantedtolearnasmuchaboutthediseaseaspossibleinordertowork
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moreeffectivelywiththeirdoctors.IdecidedtocompletelyrewritetheSclerodermaFAQinorder
totrytoproduceacomprehensivedocumentthatwouldbridgethegapbetweentechnicaland
medicaljargonfoundinresearchpapersbyexplainingeverythinginawaythatpatientswithouta
medicalbackgroundcouldunderstand.
ThefirstdraftofthecompletelyrewrittenSclerodermaFAQwascompletedinlate2013afterabout
200hoursofresearchandwriting.ThisinitialdraftoftheupdatedFAQwas34pageslong(the
existingversionwasabout8pageslong).OverthenextsevenmonthstheFAQwentthroughmany
revisionsbasedonexcellentfeedbackfromtheSclerodermaFoundationandseveralphysicians,
includingonewell-respectedsclerodermaexpert/researcher.
InApril2014IresearchedandwroteacompaniondocumenttotheFAQtitledtheGuideforNew
andFuturePatients.Ialsorealizedatthispointthatinadditiontotheseinitialtwodocuments,
therewereanumberofotherarticlesthatIwantedtowrite,includingadditionalpatienteducation
documentsbutalsoaseriesofTechnicalArticlesthatwouldcovercertaintopicsinmoredetailand
includeselectiveresearchcitations.Someofthesetechnicalarticleswouldbetargetedat
physiciansandresearchersinadditiontomoresophisticatedpatients.Asaresult,inMay2014,I
madethedecisiontolaunchacompletenewwebsitecalledtheSclerodermaEducationProject
whichwouldhouseallofthesedocuments.Thenewwebsite,locatedatSclerodermaInfo.org,was
launchedinJuly2014.
TheSclerodermaEducationProjectwebsiteisnotmeanttoreplaceorcompetewithexcellent
websitesprovidedbytheSclerodermaFoundation,theMayoClinic,JohnsHopkins,etc.Itprovides
additionalandmoredetailedinformationaboutavarietyoftopicsthatareusuallynotcoveredon
thesewebsites,forexample,detaileddiscussionsonANAandantibodytesting,informationonthe
new2013Guidelinesforsclerodermadiagnosis,etc.,alwayswrittenatalevelthatpeoplewithout
medicaltrainingcanunderstand.Mybeliefisthateducatedpatientsarebetterabletoworkwith
theirteamofphysicianstomakethebestpossibledecisionsabouttheirownhealthcare.
Update3/10/16:TheSclerodermaEducationProjectLtdisnowa501(c)(3)taxexempt
organization.ThismeansthatanydonationsarenowtaxdeductibleintheUS.
Scleroderma Patient Advocate
OncetheoriginalversionoftheSclerodermaFAQwaspublishedonlinein1996,Ialmost
immediatelybegantoreceiveoccasionalemailswithfollow-upquestionsaboutsclerodermafrom
patientsaroundtheworld(myemailaddresswasincludedintheFAQ).Forthemostpart,these
typesofquerieswerelimitedtoafewtimesamonthpriortothelaunchofthenewwebsite.
AfterthenewSclerodermaEducationProjectwebsitewaslaunchedinJuly2014,Ijoinedand
becameaveryactivememberofabout12scleroderma-relatedpatientsupportgroupsonFacebook
aswellastheInspiregrouprunbytheSclerodermaFoundation.Asaresult,overthelastyearand
ahalfmypatientsupportactivitieshaveincreasedsignificantlyandInowspendatleastacoupleof
hourseachdayworkingwithpatientsboththroughsupportforumsaswellasonanindividual
basis.IalwaysmakesurethatpatientsknowthatIamnotaphysicianandcannotoffermedical
advice.However,Iamoftenabletoprovideinformationtosclerodermapatientsthatcanbeuseful
inhelpingthemtoworkmoreeffectivelywiththeirteamofphysiciansingettingquicklyand
accuratelydiagnosedaswellastomakebetter,moreinformeddecisionsontheirownindividual
healthcare.
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Scleroderma Researcher
Inearly1993,afterreviewingalloftheresearchliteratureonsclerodermathatIcouldfind,I
developedanewhypothesisthatsclerodermasymptomsmaydevelopbeginningwithdamageto
thevascularsystemthatisadirectconsequenceofredbloodcellsbeingabnormallyclumped
together(hyperaggregation).Ifthishypothesisiscorrect(anditcaneasilybetestedbyresearch),
thenANYtreatmentthatcanbreakaparttheclumpedredbloodcellsandkeepthemdisaggregated
shouldpreventanyfuturesymptomdevelopmentandallowthebodytopartiallyorfullyhealover
time,totheextentthatthisispossible.Obviously,ifstartedlateinthediseaseprocess,complete
reversalofdamagewillnotbepossible.Plasmapheresistreatmentshavebeenshowninanumber
ofresearchstudiestobeeffectiveindisaggregatingredbloodcellsandreturningoverallblood
viscositytonormallevelsinsclerodermapatients.Therearealsoseveraldrugsthatmayhave
potentialaswell,buthavenotyetbeentestedasatreatmentforscleroderma.IntheAdditional
ArticlessectionoftheSclerodermaEducationProjectwebsite,thereisatechnicalarticletitled
“SclerodermaHyperviscosity:ImplicationsforTreatmentandResearch”thatexploressomeofthe
researchbehindthisproposednewdiseasemodel.
Inanefforttoadvancetheresearchtotestoutthispotentialnewdiseasemodelandtreatment
approach,Iamnowworkingwithseveralresearchersonaseriesofresearchpapersand
presentations.Thefirstofthese,areportonmyownverylongterm(22plusyear)experienceasa
sclerodermapatientreceivingregularplasmapheresistreatments,waspresentedasaposterata
medicalconferenceinOctober2015(Ididthepresentation)andtheabstractispublishedinthe
September2015specialissueoftheresearchjournalTransfusion.Thetitleofthatabstractis
“BenefitofLong-TermTherapeuticPlasmaExchangeTreatmentinaPatientwithCRESTSyndrome
(LimitedSystemicScleroderma):A21-yearSuccessStory”.ThenewsorganizationScleroderma
Newsdidagreatstoryonthiscasereport:
http://sclerodermanews.com/2015/12/03/scleroderma-patient-long-term-remission-tpetreatment/.
Update5/27/16:Anexpandedversionofthecasereporttitled“SuccessfulLong-Term(22Year)
TreatmentofLimitedSclerodermaUsingTherapeuticPlasmaExchange:IsBloodRheologythe
Key?”wasjustacceptedforpublicationintheresearchjournalClinicalHemorheologyand
Microcirculation.Themanuscriptversionofthiscasereportcanbefreelydownloadedhere:
http://www.sclerodermainfo.org/pdf/CHM-Case-Report-Manuscript-US.pdf.
Ipresentedasecondposter,titled“TherapeuticPlasmaExchangefortheTreatmentofSystemic
Scleroderma:AComprehensiveReviewandAnalysis”,attheAmericanSocietyforApheresis2016
annualconferenceinMay2016.TheabstractofthisposterispublishedintheJournalofClinical
Apheresis.Thisisacomprehensivereviewarticlethatexaminesalloftheresearch(whichis
actuallyquiteextensive)donetodateontheuseofplasmapheresis/therapeuticplasmaexchange
totreatpatientswithsystemicscleroderma.Hereisalinktotheanextendedversionoftheposter
thatwashandedoutattheASFAconference:http://sclerodermainfo.org/pdf/ASFA-Handout.pdf.A
fullversionofthisposteriscurrentlyinpreparationforjournalsubmissionthissummer.
Ijustsubmittedanabstracttitled"TherapeuticPlasmaExchangefortheTreatmentofSystemic
Scleroderma:AComprehensiveReviewandAnalysis"totheAmericanCollegeofRheumatology
meeinttobeheldNovember2016inWashington,DC.
Thefourthpaper,tentativelytitled“SclerodermaBloodRheology:ImplicationsforTreatmentand
Research”,willbeanexpandedandupdatedversionofthehyperviscositytechnicalarticleonmy
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website.Itwillexaminealloftheresearchthatdiscussesabnormalbloodrheologyinscleroderma
patients,includingredbloodcellhyperaggregation,andlookathowthismightleadtothe
developmentofsclerodermasymptomsaswellasexploringpotentialoptionsfornewtreatmentsif
thisdiseasemodelisproventobecorrectbysuitableresearch.
Asanon-physician,ithasbeenamajorchallengeformetogetacceptedassomeonewhomay
actuallybeabletohelptoadvancesclerodermaeducationandresearch.Iamnowfortunate
enoughtobeworkingcloselywithseveralresearchersthatnowagreethatmyproposednew
diseasemodelmaywellbecorrectanddefinitelyneedstobestudiedthroughappropriateresearch.
Thebottomlineisthis:ifmyproposednewdiseasemodelsturnsouttobewrong,thensome
researchmoneywillhavebeenwasted(whichhappensallthetime);however,ifitturnsouttobe
correct,itmayquicklyleadtonewtreatmentapproachesforsclerodermathatcouldbemore
effectiveandlesstoxicthancurrent,largelyineffective,treatmentapproachesthatareheavily
focusedonusingimmunosuppressantdrugstosuppresstheimmunesystem,frequentlyleadingto
majorsideeffectsandcomplicationsfromlong-termusage.
About Me
AfewyearsagoI“retired”fromalongcareerasacomputersoftwaredeveloper(since1964)and
successfulserialentrepreneur.Iamalsotrainedasaresearchclinicalpsychologist.Iammarried
toaphysician(whichhasbeencriticalinmyjourneyfrompatienttoresearcher).Iamaskilled
amateurmusician(clarinetandbassoon)andplayinalocalcommunitybandandorchestra.
Directlyrelatedtomyinterestinmusic,Iamalsointhemiddleoftrainingtobeacomposerof
classicalmusicfororchestra,band,andchambergroups.(Ihavecompletedthreecompositionsand
wouldbehappytosendlinksifanyonewouldliketohearthem.J).Mymainhobbiesaretennis(I
playatleastonceaday)andcooking.
©Copyright2000–2016.EdwardS.HarrisAllRightsReserved
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