AL Amyloidosis - a bone marrow related cancer

From my Sisters perspective, as a primary carer.

Everybody’s journey is unique, everyone reacts to medications &treatment procedures differently. This is about my brother’sjourney with Cardiac (AL) Amyloidosis through my eyes as one of hisclosest advocates. I share this story in hope that it may helpothers; especially if you are like me and are wanting to help &support your sick loved ones too. The bottom line is never give-up,there is always hope.

My Name is VeronicaPearce. I’ve been living in the USA as an expat Australian since1999. In October 2015 I was on a morning walk in San Antonio(Texas), when I received a phone call from my brother Lyall Pearcewho was living in Adelaide, Australia. He told me his medical testresults were back and his doctors suspected he had some form ofMyeloma that was adversely affecting his heart. I stopped walking,sat down on the side of the road, he explained it as a blood/bonecancer and said they had a few more tests to perform and we wouldknow more within the few days.

Sure enough, severaldays later Lyall’s formal diagnosis came back as Cardiac (AL)Amyloidosis which is a complication of Myeloma. Icouldn’t even pronounce these words, let-alone understand what theymeant. After some research online I learnt myeloma is a form of bonemarrow cancer where the plasma cells (produced from the bone marrow)are faulty. In Lyall’s case these faulty cells produce antibodiesthat misfold into a plaque-like protein (amyloid) that graduallydeposit in the heart tissue, slowly thickening his heart walls,adversely affecting his heart – hence the diagnosis of Cardiac (AL)Amyloidosis.

In the year prior tohis diagnosis Lyall knew something was medically wrong, his cardiacfitness was deteriorating quickly and significantly, he was findingit harder and harder each day to ride up hills on his normal 20kmround trip daily cycle to & from work. At the time he wasdiagnosed (October 2015), his prognosis was terminal, and he wasgiven 18 months to live.

The windy road wefollowed through the progression of Lyall’s treatments …

Despite a poorprognosis, Lyall’s medical team established the best way forwardwas a treatment regime of firstly chemotherapy shortly followed witha Stem Cell Transplant to remove the faulty plasma cells in hope toextend his life.

In the days &weeks following Lyall’s diagnosis we had so many questions. It wastotally overwhelming. The best thing I did at that time wasself-education: reading hospital issued brochures, literature fromthe Leukaemia Foundation, online research, forums, and lots ofquestions to Doctors, staff at the Foundations, other patients we metin waiting rooms and anyone else who could offer any insights &guidance. Much of the documentation I read initially I couldn’tunderstand, all the medical terms and complexity of it all was liketrying to read something in a foreign language. But as I graduallylearnt what the terms meant (Google Search became my best friend)things started to make a little bit more sense.

We learnt there weresome critical parameters we needed to start following to monitorLyall’s health. Like someone with Diabetes who closely monitortheir blood sugar levels and they take corrective action when its lowor high. For Lyall’s Cardiac Amyloidosis the main parameters wereinitially: Lambda Free Light Chains (FLCs), Troponin,NT-ProBNP, Temperature, Blood Pressure, Heart Rate and Weight. Oncewe understood that these critical parameters were indicative ofLyall’s future health, we took responsibility to track them andproactively took quick action &/or asked for advice/help from hismedical team when it was clear something was trending off-track.

I just want tore-iterate this, because it is really important, if a healthparameter is going off-track, or a new symptom develops, don’t waitto see what happens, act FAST and ask for advice/help. If somethingreally is wrong, there is a much better chance of recovery if it isidentified and treated early.

In October 2015Lyall started his first chemotherapy regime with Velcade(tm), also referred to as CyBorD. It was administered byintravenous infusion, treatment continued through to late December2015. During that time, the regime was successful at reducing hisFLCs down from 65mg/L to 32mg/L (normal is between 5.7 - 26mg/L). His levels weren’t quite within normal range yet, but they were atleast heading in the right direction, so in early January theyproceeded to collect his Stem Cells in preparation for his Stem CellTransplant which was scheduled for later that same month.

Despite loweredFLCs, his heart parameters were still deteriorating. Two mainparameters being followed were NT-ProBNP and Troponin, both wereescalating well above normal range. Overall, his cardiac health wasdeemed too poor for him to survive a Stem Cell Transplant procedure,so in late January 2016 the procedure was cancelled. The only wayforward was to pursue further chemo treatments and hope his hearthealth would improve at which time they would reconsider proceedingwith the Stem Cell transplant.

During a month breakfrom chemo it was hoped the Velcade regime would continue to reducehis FLC levels, or at least hold them steady for a while, butunfortunately his FLCs shot back up to 64mg/L – well above normal. Given this regime did not ‘hold’, and the heart parameters werenot improving, a different regime of Melphalan would be administeredto see if it would work any better.

After one month itwas clear the Melphalan regime was not dropping his FLCs down much atall, and we were given the choice to try it a little longer, orrevert back to the Velcade regime to quickly get his FLCs back tonormal. We decided to revert back to Velcade, this time it wasadministered by subcutaneous injections into the stomach. Afteranother three weeks his FLCs were still not dropping so Thalidamidewas added to the Velcade regime and they also changed it to beadministered by intravenous infusion instead of subcutaneousinjections into the stomach.

By late March 2016tingles in the feet & hands started developing along with dizzyspells & puffy skin from fluid retention in the body (oedema). The dizzy spells were most noticeable when transitioning from lyingdown to standing. We since learnt these are typical symptoms ofneuropathy & the oedema is typical for patients withheart-failure.

In early April 2016his FLCs had successfully reduced to 23mg/L, within normal range. Wecontinued with this chemo regime though end June 2016. However, hisheart parameters (Troponin & NT-ProBNP) were not improving makinga Stem Cell Transplant further and further out of reach. We neededto find a treatment that would not only help control his blood cancerbut would help his heart too.

Given Lyall’scondition crossed two disciplines of medicine, Hematology for thebone/blood cancer, and Cardiology for heart failure, it was becominga delicate juggle on how to treat and medicate both. There weren’tany regimes on the PBS List to help remove or reduce depositedamyloids from the blood or heart. Lyall’s options for treatmentwere becoming limited.

We asked about thepossibility of a heart transplant in the future should his heartcontinue to deteriorate, but we were told at this time it was not anoption as treatment with traditional Chemotherapy was an ExclusionCriteria for Heart Transplant patients; most chemotherapy medicationsinterfere with the immunosuppressant medications used to preventorgan rejection after transplants. Further, Lyall’s blood cancerwould probably eventually infiltrate and damage the new heart too. So, for Lyall’s condition, based on the treatments available at thetime, this was not an option. It made sense, we understood why, andat that time as hard as it was we accepted it, but it was trulydevastating trying to accept the finality of it all.

Despite Lyall’spoor prognosis, we knew the technological developments in thismedical field were moving fast – so there was still hope. Weneeded to refocus on the hope, push-on and keep exploring options.

In mid-April 2016some online research uncovered a promising Clinical Trial reportabout the use of antibiotics, specifically Doxycycline, where itdisrupted amyloid production by preventing fibril (amyloid)formation. We asked his medical team about it and they agreed it wasworth a try, so Lyall started taking Doxycycline too in addition tohis chemo regime.

In May 2016 Lyallhad a set-back, he presented to the Emergency Ward with an elevatedtemperature and feeling weak. The big lesson for us here, we waitedtoo long to seek medical attention when a key parameter wentoff-track. He was admitted to ICU with Viral Pneumonia. DuringMay/June his heart parameters deteriorated significantly. HisNT-ProBNP skyrocketed reaching over 17,000 ng/L (normal is less than900 ng/L). Lyall’s heart Ejection Fraction dropped to 32%, meaningonly 32% of the total amount of blood in his heart’s left ventriclewas being pushed out with each heartbeat (Normal is greater than55%). However, to our surprise his Troponin levels dropped back intothe normal range, and his FLC levels dropped significantly down to6mg/L, on the low-end of Normal scale too, which meant chemo could bestopped for a little while; a break from chemo his body reallyneeded.

By this time, end ofJune 2016, Lyall’s tingly fingers & feet had developed intofull numbness of limbs below the knees and numbness in the hands,including loss of ability to feel the difference between hot &cold. His dizzy spells had developed into regular and problematicblackouts with his baseline blood pressure now consistentlydangerously low. He was diagnosed with both peripheral &autonomic neuropathy, the latter causing postural hypotension, whichis when the body becomes unable to self-regulate blood pressure sowhen he stood up from a lying-down or seated position, his bloodpressure couldn’t auto-correct quickly enough to sustain adequateblood to the brain, so he would blackout and collapse. Fortunately,they could medicate to help raise his baseline Blood Pressure to helpreduce the blackouts, but there was nothing they could do to helpwith the numbness in the limbs. Velcade & Thalidamide could nolonger be used for Chemo due to these adverse side-effects. We hadto find another treatment.

While he was on abreak from Chemo we took stock of our options, I knuckled down anddid more research. I called almost all the Amyloidosis Centresthroughout Australia and the USA, including the Stanford AmyloidCenter and Mayo Amyloid Clinic in Minnesota, asking for educationalliterature & advice. I read abstracts & papers from bothClinical Trials and medical conventions specialisingin Amyloidosis treatments & future developments. The mostpromising treatment found from this research was daratumumab(Darzalex), it was the first monoclonal antibody approved for use inmultiple myeloma, and a small Clinical Trial in the USA showed reallygood results for Cardiac (AL) Amyloidosis patients. PreliminaryTrials were showing a Haematological Response rate for Single Agentdaratumumab of 90% (n=18, CR=6, VGPR=3, PR=7, NR=2)1,2compared to most other agents that barely reached 70%. We approachedLyall’s medical team requesting we try daratumumab, they advisedthey had been monitoring the development of this new treatment too,but unfortunately, daratumumab was only available in the USA and notavailable in Australia yet. Our team agreed to continue to closelymonitor daratumumab’s entry into the Australian market, but weneeded to find something else for now instead. We briefly consideredtravelling to the USA for treatment with daratumumab, but it wasclear this would be cost prohibitive (that’s a whole other story)and most likely a poor quality of life choice with Lyall beingseparated from family & friends. Our remaining choices withinAustralia were to either select another Amyloidosis treatment regimefrom the PBS List, or find a Clinical Trial for a new treatmentregime. My research continued.

The next best thingwe found was a Clinical Trial for a new promising monoclonal antibodytreatment called PRONTO NEOD001. It was specifically for treatment ofCardiac (AL) Amyloidosis. Preliminary results showed it hadpotential to stop formation and even remove amyloid deposits from theblood and heart. The medical team agreed to help us pursue thisClinical Trial and helped us work through the process.

Over the next fewmonths Lyall’s FLC’s were fortunately holding firm in the normalrange, so the pressure was off to find another quick Chemotherapysolution. Instead, this gave his medical team time to get things setup for the NEOD001 Trial which was being managed in Melbourne. Ittook many months of paperwork, interviews & preliminary medicaltests to qualify for the Clinical Trial and much time and effortinvested by both his Adelaide medical team and the Clinical Trialteam in Melbourne to coordinate everything.

While we waited forthe approvals to participate in the PRONTO Clinical Trial, wecontinued our research and we found another medication called EGCG. It showed promising preliminary Clinical Trial results to help removeamyloid deposits. Given EGCG was readily available, we asked hismedical team if we could try it while we waited to get approvals backfrom the PRONTO team. Although they were against it and erring onthe edge of saying ‘no’, they finally relented and agreed to letus try it as an interim treatment. (In hindsight, they were right,the EGCG regime did not help Lyall’s condition at all).

After much waiting,in early January 2017 Lyall was invited to travel to Melbourne by thePRONTO NEOD001 team for his final interview and medical tests, thelast step to quality for the Clinical Trial. We were excited. Reports were showing good preliminary results and it was wonderful tohave hope again. Lyall’s FLC levels had crept up over the last fewmonths and was bordering the top-end of the normal range, so it wasperfect timing for the PRONTO NEOD001 regime to commence. But wereturned from Melbourne without qualifying for the trial due to asilly mix-up on our end. We were told Lyall was to stop taking ALLmedications for 6 weeks prior to this interview, but we interpretedit as to stop taking Chemotherapy. Although Lyall had beenchemotherapy free for months, he had still been taking EGCG andDoxycycline, which disqualified him. This was a BIG lesson for us;to make sure we clearly cross-matched medications, one-by-one, withhis medical team regularly to ensure we were using the rightmedications & doses. Although Lyall missed out this time, he wasinvited to qualify in 6 weeks’ time if the last spot in theClinical Trial had not been filled by someone else. In mid-Februaryhe travelled back to Melbourne again, this time he qualified and wasapproved to participate in the Trial. Two weeks later (early March2017) he was given his first infusion which had a 50/50 chance ofbeing either NEOD001 or a Placebo. It was a randomized, double-blindtrial, so we would never know if he was being given NEOD001 of thePlacebo. Risky, yes, but with virtually no other options offeringreal hope, it was worth the risk at that time.

Ever since Lyall wasdiagnosed, to keep ourselves up-to-date on the medical industrydevelopments to treat Lyall’s condition, we signed up to receiveemail updates, newsletters and media alerts from variousorganisations, institutions, foundations & medical manufacturerswho specialized in Amyloidosis. These updates were particularlyuseful. In July 2017 we received an email alert advising us thatdaratumumab had now been approved for use in Australia on the basisof Compassionate Access only (but it was still not on the PBS). Atthe time, Lyall’s FLC’s and amyloid deposits were not showing anysigns of improvement from 4 months of treatment on the NEOD001 trial,so this alert on daratumumab was a new sign of hope. We immediatelyreached out to his medical team, stating we still wanted to pursuedaratumumab as a high priority, and asked what needed to happen nextto get the ball rolling. His team reported they were aware of theannouncement and they had already commenced correspondence with themanufacturer and discussions were in progress, but it would taketime.

By mid-September2017 Lyall was still deteriorating, and his FLC levels were stillincreasing at a steady rate and were now well above normal. He optedto abandon the PRONTO NEOD001 clinical trial and as a stopgap herecommenced Doxycycline & EGCG until another chemo regime couldcommence.

Looking at theremaining chemo regimes that were left for Lyall to try on theAustralian PBS list, it was quite disheartening as they were allderivates of treatments he’d tried in the past which were risky dueto adverse side-affects, or they were derivatives of treatments thathad not worked. In desperation, knowing daratumumab was now inAustralia, and it wasn’t clear the status of getting Lyall accessto it, I reached out for help to the medical manufacturer directly,the hospital, various local, state & national health ministers. I let his medical team know my actions and they promised they werestill vigorously pursuing access to daratumumab and it would still bea long road ahead before we could get it, but the wheels were slowlyturning and progressing. We just needed to wait and hope we couldget it before it was too late. We had to find something else in themeantime.

In early October2017 he stopped EGCG & started a new low-dose regime of chemocalled Lenalidomide (Revlimid). The new regime was one of very fewleft on the PBS List, all of which were undesirable based on Lyall’smedical history. Fortunately, it successfully lowered Lyall’s freelight chains down to normal levels within a couple of weeks and hisside-affects were minimal. In November 2017 chemo was intentionallypaused to see if these low FLC levels would ‘hold’. They didn’t. This was incredibly upsetting as this meant Revlimid was not goingto be sustainable for Lyall for very long. Revlimid was re-startedat half the low-dose in hope it would buy us more time, and it wasback to the drawing board to determine what regime to try next. Inthe background, his medical team continued to pursue getting accessto daratumumab.

By December 2017Lyall’s FLCs had slowly crept up to above normal levels again andby June 2018 it was clear they were going to continue rising. Halfthe low-dose Revlimid was no longer sustainable and an increase indose was too risky due to adverse side-effects. In June 2018 Lyalltook his last dose of the Revlimid chemo regime.

In mid July 2018 toour complete surprise, Lyall’s medical team announced they hadfinally secured special access to daratumamab (Darzalex) and thatsame week he had his first infusion. Within 3 weeks his FLC levelsdropped back to normal. Lyall has been on daratumumab ever since(its now February 2021 as I write this article) and his FLCs havebeen maintained at normal levels with no other adverse side-effects. He also continues to take Doxycycline.

Back to late 2018,although on a Haematological basis Lyall’s FLCs were now beingmaintained at normal levels using daratumamab, and by definition inmedical terms he was (and still is) considered to be in ‘remission’, but it is still the view of his medical team he still has cancer andtreatment needs to continue. Even though his FLCs are at normallevels, its still unknown what percentage of these FLCs are ‘faulty’and could still be misfolding into amyloids and subsequentlydepositing in his heart. Lowering the FLC levels in the blood couldsimply be lowering the ‘rate’ of amyloid depositing in the heart. Its incredibly hard to measure.

Even though Lyall’sFLCs levels had been stabilized, his heart was now too severely andirreversibly damaged and Congestive Heart Failure was stillprogressing. In late 2018 we needed to shift gears and move ourfocus from the Hematology discipline and start focusing heavily onthe Cardiology side of things. Back to research, asking lots ofquestions & more self-education. There were so many differentheart devices and aids that can be used to assist cardiac patients. Lyall’s medical team helped us understand all the options availableto Lyall to potentially improve his heart condition. In February2019 Lyall had heart surgery to have a bi-ventricular deviceinstalled.

By December 2019Lyall was still stabilized on a haematological front with normal FLClevels, but his heart condition was still deteriorating with hisEjection Fraction getting worse. His fluid retention (oedema)becoming almost unmanageable at home, requiring frequent in-patienthospital care and he was experiencing constant low blood pressure andblackouts were becoming more and more frequent. Again, we startedasking his medical team for possible options moving forward. What dowe do now?

The biggest questionwe asked was associated to a Heart Transplant. We knew that Lyallwas previously excluded from a Heart Transplant because hischemotherapy medications would interfere with the immunosuppressantmedications used to prevent organ rejection after the transplant. But Lyall was no longer on chemotherapy medications, instead he wasnow on a new monoclonal antibody regime (daratumumab). Would themonoclonal antibody interfere with the transplant immunosuppressantmedications too? If not, does that open the door for Lyall to now beconsidered for a Heart Transplant? Also, knowing that his FLCs havebeen maintained at normal levels now for a significant time (1.5years), medical remission, does this also change things with regardsto qualifying for a Heart Transplant? His medical team said this nowlies in a ‘grey area’ and would need to be assessed on acase-by-case basis. Again, Lyall’s medical team agreed these weregood questions and promised they would source answers, but likebefore it may take some time.

Time passed, Lyallstruggled through and was still deteriorating from Congestive HeartFailure. Then in early 2020 Lyall was approached by his medical teamwith an offer to participate in a Clinical Trial for “Orthotopicheart transplantation followed by autologous stem celltransplantation in patients with cardiac AL amyloidosis - a Phase IIstudy”. Words can’t explain our relief – once again there wasrenewed hope. Over a period of about 2 months, all the preliminarymedical tests were done to determine whether he would qualify for theHeart Transplant & Stem Cell Transplant (HT/SC) Clinical Trial. Many medical tests later and several trips to Sydney for interviews,he was told he qualified. We were advised he was now added to theHeart Transplant waitlist. What an incredible turn of events.

In May/June 2020Lyall’s hospital visits became so frequent due to unmanageablefluid retention and blackouts that he was spending more time inhospital than at home. By mid-June he was deemed to have anunmanageable cardiac condition by Royal Adelaide Hospital and he wasMedivac’d from Adelaide to Sydney for more specialized treatment atthe St Vincent's Hospitalwhich is a leader in Cardiac Medical Services & Care.

At the end of June2020, Lyall’s heart could no longer pump enough blood through hisbody to sustain him and some of his other organs started to fail. With his heart being abnormally shaped and with very thick walls,traditional cardiac aids to improve his condition would require novelsurgery and was deemed too risky. Instead, Lyall became the firstperson in Australia to have a tiny temporary pump called an Impella,installed inside his heart to help boost his blood flow to keep himalive while he continued to wait for a donor heart. The life-savingImpella surgery sustained Lyall for 4 days until he received a HeartTransplant on 1st July 2020.

Once we were adviseda donor heart had become available, everything happened so fast. Theemotions were overwhelming; gratitude, relief & joy this day hadfinally come, fear & worry of what could go wrong and theunknown, grief & sadnessknowing another family’s lives had tragically changed forever.

The Heart Transplantsurgery itself only took 5 hours. Lyall was conscious again within24 hours following surgery, and with help he was able to stand-up andwalk a few steps within a couple of days. His recovery over the nextweek was incredible to watch and much faster than I ever imagined.

But then into thesecond week of recovery we had a major set-back, he started to feeloff-colour, brain fog and confusion, difficulty with handmotor-skills (dropping things), balance, weak, affected vision (hesaw sparkly little worms in the clouds) and he had a few unusualheart-beat events (fibrillation). A few days after he had anexternal pacemaker removed he collapsed. His medical team actedincredibly fast and he was in open heart surgery within minutes tofix the problem. He had suffered an internal bleed in his chestcavity which caused a Cardiac Tamponade event; where the blood filledhis chest cavity, pressurised it, and the pressure caused his newheart to fail.

His recovery startedover and again. He was conscious within 24 hours of the secondsurgery. But this time, something was slightly different, his speechwas slow, somewhat simple and disjointed. Then he’d snap out of itand speak normally, then he’d revert back and speak slow again. Ididn’t think anything of it at first and put it down to ‘recovery’,but after a quick consultation with family we felt it critical to letthe medical team know in case he’d had a TIA (mini stroke). Theyacted immediately and he went through many tests. They all came backclear, no evidence of a stroke.

But his behaviourwas still not normal and it was getting worse. Confusion, speech,vision, hearing, motor-skills were all being affected in waves. Forone hour he’d be ok, then for 1-2 hours he’d fall into a statewhere he could not function & he was reporting that he was inpain. Then he’d snap out of it and be normal, no pain. The cyclecontinued and the episodes became longer and worse. Specialists werequickly brought in from many disciplines throughout the hospital toassess and run tests to rule out various potentially debilitatingdiseases, viruses and disorders. All tests came back clear. It wasduring this time, when he had lost his ability to effectivelycommunicate, where I felt I was advocating for him the most. KnowingLyall’s normal behaviour, it was easy for me to see when there wasimprovement, deterioration, change &/or new symptoms. Often, Iwould see changes quicklyand could raise the red flag early. His nurses & carers couldn’tto do this, because they didn’t know Lyall and couldn’tdifferentiate normal behaviour from abnormal. I would try to makesure I was visiting Lyall during the times the Doctors would do theirrounds, and I would respectfully give my bedside observations to hismedical team & try to keep updated on his progress and nextsteps. They in turn would help guide me on behavioural things towatch-out for and what to take seriously and what to ignore. Hismedical team made me feel like an important part of their team andoften acted, ordered tests, if I’d raised a concern about a newsymptom. They listened to my concerns and briefly looked at my dailynotes (I’d have to keep these brief otherwise they wouldn’t readthem through). After all serious diseases, viruses, disorders andneurological conditions were ruled out, he was diagnosed withDelirium. It then became a challenge to determine what was causing itso they could pull him out of it. Every day they changed somethingto see if it would help and I’d provide bedside feedback on whetherit had made a difference or not. Then the next day they’d tryagain, and again, and again. One evening when I was looking backthrough all my notes, it occurred to me that his condition started todeteriorate about the same time they had changed one of hisanti-rejection medications, which happened several days prior to hisCardiac Tamponade event. I read the symptoms of possible allergicreactions to this medication, and they seemed similar to what Lyallwas experiencing. By this time is was 2am, so I phoned the hospitaland reported this to the Doctor on Duty, then met with his normalmedical team the following morning to discuss further. By that time,they had already changed his anti-rejection medication, they’d alsoordered infusions of immunoglobulin as well as a few other minormedicine changes. The following morning, I walked into see Lyall,and to my complete surprise he was sitting up in his bed playingcards and simply said “Hi V”. He’d totally come out of hisDelirium. To this day his medical team are still not sure whatcaused it, there are several theories and differences of opinions,but ultimately they believe it was a chemical imbalance in the brainthat could have been a result of his new medications (or combinationthereof) and his compromised immune system. It took three weeks forhim to fully recover. Lyall doesn’t remember the Delirium, herefers to this period of time as his visit to “La La Land”; oneday his fingernails were short, the next day he woke-up and they werelong.

Seeing a loved onego through this was totally heart wrenching and incredibly scary. Itwas made worse being in the middle of Covid and having all theborders closed which stopped other family members from travelling tohelp support us. But the medical professionals never gave up andkept giving me hope that they would work it out. And they did.

After weeks of rehabfor severe muscle atrophy Lyall was discharged from hospital. He wasrequired to stay locally in Sydney as an outpatient until he’dcompletely stabilised on theanti-rejection medication and been given the green-light to travelhome to Adelaide. That green-light came at the end of October 2020,a few weeks after the border between NSW & SA had reopened. Perfect timing to avoid complications of Quarantine.

Upon returning toAdelaide Lyall was able to settle back into his own house &routine with his family and continue focusing on rehab. We had alovely Christmas together with all our extended family. Then inJanuary 2021 the preparations ramped up for his next procedure, theAutologous Stem Cell Transplant.

As I write thisarticle (its mid February 2021) Lyall is still in hospital and isalmost 2 weeks into the Stem Cell Transplant procedure. As I didbefore, I elected to travel with Lyall to Sydney as his advocate. This whole procedure has again been tough on Lyall as a patient,simply because it makes people feel so sick and weak. But we knew itwould be hard for him going into the procedure. Despite Lyallfeeling so sick, everything has gone to plan so far without any majorcomplications. I visit Lyall morning and night and try to keep trackas best I can on the parameters that are important and keep hisfamily updated in Adelaide. If I need help understanding something,I ask his nurse or medical team questions. But I make sure to bepolite, not too demanding, non-judgemental and sensitive to theirtime as often they have a lot on their plate, and I don’t want tobecome a nuisance. My intent is to advocate, support and help –not to make life difficult or unpleasant for anyone. We are allthere for the same reason, to help Lyall. If I see something thatconcerns me, I alert a member of his medical team. If I know anon-routine procedure or medication is scheduled, I follow-up to makesure the wheels are turning and its on track to occur. If I suspectsomething has been overlooked or missed, I alert someone – just incase. We’re all only human after all and we need to have eachother’s back, help each other for the good of our common goal.

Lyall’s on therecovery side of the curve now, with his immune system ramping upproduction of new white blood cells from his Stem Cells that wereharvested back in January 2016. He is scheduled to be released fromhospital in two days, followed by another two weeks in Sydney as anout-patient under observations before he is cleared to travel backhome to Adelaide.

Lyall’s overalljourney has entailed a large range of different treatment options,with varying degrees of success, including participating in multipleClinical Trials. These leading-edge medical developments offeredhope at times when there were no other treatment options availableand fortunately for us several of them delivered. From Lyall’sendeavours over the last 5 years, support from his advocates, andfrom an incredible & progressive team of medical experts, he’smade it through and potentially forged new pathways for successfultreatment of others who have the same diagnosis. An extended life,long term maintenance of his condition, and even a cure is now a realpossibility.

To have Lyall stillwith us today in our view is a medical miracle. I can’t explainthe emotions felt by our family and how grateful we are – therejust aren’t any words.



Complete Response (Hematologic Response to Treatment Regime).Measured in Number of Patients in the Study.
Lambda Free Light Chains, proteins made by plasma cells, a type ofwhite blood cell.
Heart Transplant
Number of Patients in the Study
Is a hormone produced by your heart which is relates to hearthealth and can detect heart failure & other cardiac problems.
No Response (Hematologic Response to Treatment Regime). Measuredin Number of Patients in the Study.
Partial Response (Hematologic Response to Treatment Regime).Measured in Number of Patients in the Study.
Stem Cell
Proteins relating to heart contractions & muscles. Hightroponin levels can indicate a problem with the heart.
Very Good Partial Response (Hematologic Response to TreatmentRegime). Measured in Number of Patients in the Study.


  1. Daratumumab Produces DramaticResponses in Relapsed Light Chain Amyloidosis

Published Online:Monday, Sep 12, 2016

Gregory P. Kaufman, MD,Hematology at Stanford University


  1. Hematologic Responses andCardiac Organ Improvement in Patients with Heavily PretreatedCardiac Immunoglobulin Light Chain (AL) Amyloidosis ReceivingDaratumumab

58th ASH Annual Meeting& Exposition, San Diego, December 3-6, 2016

Myeloma: Therapy,excluding Transplantation

Program: Oral andPoster Abstracts

Gregory Kaufman, MD1,Ronald Witteles, MD2*, Matthew Wheeler, MD3*, Patricia Ulloa, RN4*,Marie Lugtu, RN4*, Sally Arai, MD5, Stanley Schrier, MD6, RichardLafayette, MD7* and Michaela Liedtke, MD6


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