By Jenny A | Posted - Jan 15th, 2020

 

 

 

 

Myeloma Highlights From ASH: Jack Aiello's Super Summary

According to myeloma patient advocate Jack Aiello (definitely not medically trained)

(Glossary of terms are below)

PREFACE

This is my 14th year attending ASH (American Society of Hematology), where over 30,000 attendees from all over the world (hematologists/oncologists, lab researchers, oncology nurses, scientists & 300 pharma companies) present the latest research results via both oral presentations (1000) as well as posters (3000) on all blood diseases, especially cancers.  This year there were more than 800 abstracts on Multiple Myeloma (MM) alone, with more than 100 of these selected for oral presentation. I’m grateful to the IMF and their pharma sponsors for sending me to ASH so that I can learn and share my patient perspective with you.

Rather than attending talks on Biology, I typically focus on the Clinical Trials, which I’m able to understand and are more relevant near-term to patients.  Even at that, there are overlapping oral sessions as well as 4’x6’ posters without reprints, so it’s always possible that I have not included something of interest to you or made a typo because I can’t read my own writing as detailed powerpoint slides are presented quickly. You might want to view the published abstracts at www.hematology.org and various press releases.  Wherever possible, I’ve listed Day-Abstract#-Lead Investigator after the trial results, e.g. {Sat-140-A.Krishnan}. Clicking on the abstract number brings up the actual abstract, though updates may have been presented at ASH.  

There are many ways to learn more about results from this conference.  There are scheduled webinars which you can listen to live or by replay. You’ll also find some patient blogs (including mine) as well as myeloma expert video interviews posted on the Myeloma Crowd (www.myelomacrowd.org), Patient Power (www.patientpower.info), and IMF website (https://ash2019blogs.myeloma.org/) among others. And all of us in the SF Bay Area should attend the LLS Blood Cancer Conference (which includes updates from ASH) Jan 25, 2020 (register at https://www.eiseverywhere.com/ehome/index.php?eventid=474144&).  Dr. Sandy Wong from UCSF will do a great job presenting the latest information. 

Presentations and posters of clinical trial results follow the same format: Background (including hypothesis), Study Objective, Design & Treatment schema, Patient Characteristics & Cohorts, Responses (include high-risk cytogenetics), Toxicity (hematological and non-hematological), Conclusion, and Next Step.  Remember, the goal of Phase 1 (typically handful of patients) is to determine “Maximum Tolerated Dose”; Phase 1/2 and 2 (typically 25-75 patients) continues to measure dosage escalation and safety while looking at responses; and finally Phase 3 (several hundred patients) compares response rates between new and current standard of care (SOC) treatments.

Treatment schedules, typically for ND (Newly Diagnosed) or RR (Relapse/Refractory) myeloma are defined for stages of Induction, and optionally Transplant (SCT), Consolidation, and Maintenance with specified Randomization along the way; dosage amounts and scheduling are provided for each drug along with optimum number of treatment cycles (typically 28 days). Risk stratification correlates various patient characteristics such as cytogentics-FISH analysis (e.g. chromosome deletions and translocations) and gene-expression profiling (GEP) but is defined differently than Risk for SMM (Smoldering Myeloma). And while all these details are provided in the actual abstract, I don’t necessarily list them below. Risk measurement in SMM evaluates the identifiable chance of progressing on to active Myeloma (High Risk) or not progressing (Average or Low Risk).

HIGHLIGHTS (e.g. My Takeaways...more details follow)

  1. I would say this year’s ASH didn’t contain any surprises. This past year only saw the approval of one new drug, Selinexor, so many of the ASH studies provided another year of data for Immune Therapies such as CAR-T, monoclonal antibody drug conjugates,  Bi-specific T-cell Engagers, and naked monoclonal antibodies. In addition, new drugs like Venetoclax, Isatuximab, and Iberdomide as well as new combinations therapies for both ND and RR patients are making their way through trials...some were discussed at ASH and some were not.
  2. Between the US and China, there are lots of myeloma CAR T trials and the patient experience numbers are starting to grow. More importantly, newer versions of CAR-T technology are focusing on producing longer responses.
  3. Several studies focus on adding Daratumumab (Darzalex) to frontline treatment, typically resulting in improved responses. Upfront studies: DVRd, DKRd, DweeklyKRd, DCVd, DIRd, DVMP. Dara really has become an excellent drug for myeloma patients and will become even easier to take when given as a 5 minute shot in the future (quite likely to get FDA approval in 2020).
  4. It’s difficult to believe but I didn’t attend a single presentation on transplants (other than when an SCT was part of a regimen being studied). Part of the reason for this is that time of the transplant sessions conflicted with immunotherapy presentations. I will try to report on what I learned about SCT’s.
  5. MRD (Minimum/Measurable Residual Disease) results are often incorporated in trial results, however the specificity of MRD varies, typically 10-5 or 10-6 or even sometimes in between, e.g. 3x10-6. In addition, imaging results (PET-CT or MRI) may or may not be included as part of patient responses.
  6. There continue to be trials that focus on specific groups of patients, such as HR SMM and HR MM. Unfit and frail MM patients are also studied in order to improve their treatment outcomes with good quality of life.

COMMENTS AND DISCUSSIONS I FOUND PROVOCATIVE

  1. For High Risk SMM patients, “Should the doctor decide treatment for patients or should patients decide after getting appropriate information?” J San Miguel (Spain)
  2. “When the M-spike increases ½ gram and hemoglobin decreases by ½ gram, that SMM patient has a 90% risk of progressing to myeloma within 2 years.” SVRajkumar (Mayo)
  3. “The older the patient, the lower the percentage of a patient being high risk.” T Martin (UCSF)
  4. “Personally, I think High Risk SMM is more like MM, and should be treated as such. Maybe one day it will be defined as myeloma and lower risk SMM will become MGUS.” MV Mateos (Spain)
  5. “Each of these therapy combinations for myeloma should be modified to the patient’s needs.” J Mikhael (TGen, CMO of IMF)
  6. “For T(11;14) patients, Venetoclax + Vel-d is dramatically helpful.” B Durie (Cedars-Sinai, CoB IMF)
  7. “One reason CAR-T’s aren’t lasting longer is that patient T-cells are exhausted. Earlier treatment might be more effective.” J Mikhael (TGen, CMO of IMF)
  8. At the IMF Symposium “Approaches to Achieve Best Possible Outcomes in MM”, about 1000 attendees (2/3 physicians) came from all over the world with a surprising majority attendance from Europe: Euro-40%, Can-24%, US 22%, Asia-9%, ROW-5%.
  9. To help determine treatment for relapsed/refractory MM patients, Dr. Rajkumar suggested using the acronym TRAP: T-Timing of relapse; R-Response to prior treatments; A-Aggressiveness of relapse; P-Performance status (e.g. what can the patient handle).
  10. “Checkpoint inhibitors for MM are not quite dead yet.” A Cohen (UPenn)
  11. Some high risk is higher risk than others. “Del 17p is high risk when occurring in >55% of myeloma cells. If TP53 mutation is also present, this patient is ‘ultra’ high risk.” J Kaufman (Emory)
  12. “For High Risk myeloma, Emory favors KRd -> SCT -> KRd maintenance, but more study is needed.” J Kaufman (Emory)
  13. For the IFM 2009 study (VRd with transplant vs. more VRd with 1 yr Maintenance), MRD negativity was higher in transplant arm (30% vs 20%). However, progression free survival was the same for MRD negative patients in both arms, reinforcing the mantra that achieving deep responses, no matter how you get there, is important. E Manasanch (MD Anderson)
  14. “The first CAR-T talk at ASH was presented in 2015 as a Late Breaking Abstract. Today we’re focused on making CAR-T’s better by 1) Developing other targets (e,g, dual targets), 2) Better product composition (e.g. bb21217 improvement over bb2121), 3) Better cell engineering (e.g. “armored” or “on-switch”), 4) Timing of treatment (e.g. give earlier) and 5) Thinking ‘Inside’ the box (better production).” N Shah (UCSF)
  15. “These products may become complimentary: CAR-T has a duration issue so add a BiTE or ADC for maintenance.” MV Mateos (Spain)
  16. I didn’t see any talks on the antibody drug conjugate by GSK2857916 (Belantamab, Belimaf), Dr Cohan reported that this drug, which previously demonstrated 60% ORR as a single agent for N=35 RRMM patients (57% more than 5 LOT), is actively being tested in DREAMM studies. DREAMM-2 study just announced 32% ORR for nearly 200 patients who were triply refractory (PI, IMID, and Dara) but about 25% of patients have ocular toxicity.

SMOLDERING MM

  1. A "preventative strategy" approach. Phase 2 trial results for Ixazomib (Ninlaro) + Rev-dex in High Risk SMM. Fifty three patients were given nine cycles of the triplet combo (4gm-25gm-40gm), then 15 cycles of maintenance (4g-15g-0 dex) is effective. sCR=31%, >= VGPR=50%, ORR=94% with 70% MRD- at 10-6 for >= VGPR patients. And to date, none of these patients have progressed to myeloma.  Longer follow-up for disease outcome is on-going. {Mon-580- M Bustoros}
  2. While the previous trial 580 would be considered a “preventative strategy”, this study is considered “curative strategy” for HR SMM patients. This GEM-CESAR regimen is KRd -> SCT -> KRd consolidation -> Rd maintenance up to 2 years. For 99 patients, overall response rate and complete response have been measured at different stages after induction (94%, 43% for 90 patients), after SCT (99%, 63%, for 83 patients), and after consolidation (100%, 75%, for 81 patients). Similarly MRD at 10-6 increases at each step 31%, 56%, and 53% respectively. And at 35 months, progression free survival and overall survival are 92% and 96% respectively.  Interestingly, the protocol was amended to rescue patients with Dara-Pom. Of course, more time is needed before it can be said if this is curative for some High Risk SMM patients. {Mon-781- MV Mateos}

FRONTLINE (INDUCTION OR FIRST LINE) THERAPY

  1. The Phase 2 study with Dara-VRd x4 induction, SCT, Dara-VRd x2 consolidation, 24 months Dara-R maintenance compares 200 randomized patients to a comparable VRd arm (called the Griffin trial). So far, the Dara arm improves response rates and depth of response (>= complete response 80% vs 61% so far...keeps improving after each phase) but 2-yr progression free survival (96% vs 89%) and 2-yr overall survival (98% vs 96%) are similar (likely due to short timeframe). The Dara arm has more neutropenia but otherwise side effects are similar.  So these results will need to be examined at 1 year and 2 year post maintenance. {Mon-691- P Vorhees}
  2. This study targets Unfit and Frail MM patients getting ixazomib-Dara-low dose dex (20mg cy 1-2, 10 mg cy 3-9, 0ngoing maintenance up to 2 years). For 46 patients (23 in each study arm), ORR was 74% in Unfit and 78% in Frail. Med PFS was 23 months and 12 months respectively. Although there was limited toxicity, some infections were noted, resulting in early mortality. {Mon-695- S Zweegman}
  3. The Alcyone study for Non-TE myeloma patients (N=706) was updated for overall survival, having randomized pts with VMP +/- Dara. At 42 mos, PFS was 36% vs 19%, ORR was 91% vs 74%, MRD- at 10-5 was 28% vx 7%, and OS was 75% vs 62%, all favoring the Dara arm. {Mon-859- MV Mateos}
  4. This Ph 2 study of weekly Carfilzomib -wKRd-Dara for 8 cycles showed that 23 of 30 pts achieve MRD- (10-5). As such, a Ph 3 trial called ADVANCE comparing wKRd-D vs SOC is forthcoming. {Mon-862- O Landgren}

TRANSPLANTS/CONSOLIDATION

  1. Although I wasn’t able to attend, this randomized Phase 2 study compared Carfilzomib-dex maintenance vs Observation after getting Cfz-Cy-Dex induction in Salvage SCT. For N=168, the Cfz-dex arms prolonged Time To Progression by 10 mos. {Mon-601- H Gregersen}
  2. This study assessed the efficacy of Elo+R+d -> SCT -> ERdx4 consolidation -> ERd-lite x 2yrs for N=52 pts. Standard risk ORR=89% (>=CR=43%) and for HR ORR=75% (>=CR=25%). HR had similar drop-offs for 18-month progression free survival (88% vs 68%) and 2-year OS (94% vs 49%). {Mon-603- J Berdeja}

TREATMENTS FOR RELAPSED/REFRACTORY (R/R) PATIENTS

  1. In the only oral abstract involving our new myeloma drug, Selinexor, in a study called STOMP, Christine Chen examined the all-oral treatment of Selinexor (Xpovio), Pom and dex for relapsed/refractory multiple myeloma who had received prior Revlimid and PI’s, SPd showed 56% overall response rate for N=51 patients averaging 4 lines of prior treatment and a median progression-free survival of 10.4 months for all patients. Expected dose of Selinexor to be 60 or 80 once weekly. {Sat-141- C Chen}
  2. This Late Breaking Abstract (which I was not able to attend) compared Dara-Kd vs Kd for RRMM patients in a Ph 3 study (N=466) called Candor. It showed that the addition of Dara decreased the risk of progression and death by 37%. And the Dara-Kd arm further produced nearly 10 times as many MRD- results at 12 months, as well as better & deeper responses ORR (84% vs 75%) and CR (29% vs 10%). {Tue-LBA-6- S Usmani}

MAINTENANCE THERAPY

  1. For 91 patients who had an SCT followed by Ixazomib-Revlimid-dex (x4) consolidation, this Phase 2 trial compared the results of randomizing patients to Rev vs Ixa maintenance. After 12 cycles, both improved MRD- (at 10-6) by 13% and 8% respectively. Side effects neutropenia, thrombocytopenia, and GI was higher in Rev but no Grade 3 in either group. While the goal of this trial was to determine non-inferiority of Ixazomib, the trial was actually stopped due to insufficient Revlimid sample size since about 1/3 of patients stopped Rev maintenance early due to toxicity. Therefore those patients intolerant to Revlimid may benefit from Ixazomib. {Mon-602- R Vij}
  2. This is a complex regimen because it uses MRD negativity to guide treatment. This MASTER trial regimen for 69 patients is Dara-KRd induction, SCT, and then consolidation of 0, 4 or 8 cycles of Dara-KRd according to MRD status at each phase of therapy. When patients reach 2 consecutive MRD- status (“confirmed MRD-“), they stop treatment; if they never do achieve MRD-, they go onto Rev maintenance. So far 81 patients have been enrolled. MRD- at the end of induction is 40%, SCT is 73%, and Consolidation is 82% but these are at 10-5. MRD at 10-6 is being explored and so far shows 27%, 47% and 63% at those same points. So far 26 patients have reached confirmed MRD- and of those tested, 17 of 17 are also PET-CT negative. This highly significant clinical trial is designed to safely get myeloma patients off of continuous therapy, something we would all like to achieve. {Mon-860- L Costa}

NEW DRUGS

  1. In a Phase 1b trial Dr. Amrita Krishna provided early results from Takeda’s new drug called TAK-079, an anti-CD-38 monoclonal antibody (like Dara and Isatuximab). For N=34 patients with dosages varying from 45mg-1200mg, the final dosage will likely be either 300mg but more likely 600mg with overall response rates of 33% and 56% respectively, and 21% of these patients had prior Dara.   An interesting difference between this anti-CD-38 mAB compared to Dara and Isatuximab is that it is an IGG lambda immunoglobulin molecule, whereas Dara and Isa are IGG kappa immunoglobulin molecules. Perhaps this may influence its effectiveness, such as maybe working on patients who relapsed on Dara or Isa.” {Sat-140-A Krishnan}
  2. Luciano Costa presented the first clinical study of a Celgene (now BMS) product CC-93269 that combines BCMA with a T-cell Engager CD3 and is a two-hour IV infusion. For N=12 getting >= 6mg, 89% achieved ORR with 44% CR/sCR. Adverse Events included 75% of patients experiencing cytokine release syndrome, mostly in first or second dose but managed with steroids or Tocilizumab. {Sat-143-L Costa}
  3. I wasn’t able to attend this talk about CLR 131, a radiotherapeutic designed to deliver cytotoxic radiation to selective cancer cells via a half-hour IV. For 16 heavily previously treatment myeloma patients, 8 achieved partial response and the rest achieve minimal/stable disease. {Sat-144-S Ailawadhi}
  4. AMG 701 is a BiTE®(bispecific T-cell engager) targeting BCMA with a significantly extended half-life which has hopefully replaced Amgen 420, a similar product but one which requires continuous infusion over 4 weeks. AMG 701 is given weekly as short-term infusions.  This study, which I didn’t not attend, was “pre-clinical” but we’ll be watching this drug closely based on the initial successful responses from AMG 420. {Sat-135-YT Tai}
  5. Venetoclax continued to show efficacy in t(11;14) RRMM patients in 2 talks. I wasn’t able to attend either but Ven+Dara+dex showed 92% ORR. In the other study Ven+dex for N=51 resulted in over 50% ORR for varying dosages. {Mon-925-N Bahlis}{Mon-926-J Kaufman}

CAR-T STUDIES FOR RELAPSED/REFRACTORY MYELOMA (ALL TARGETING BCMA UNLESS NOTED)

  1. China presented LCAR-B38M updated findings of their Legend-2 for 57 patients with 25 month follow-up. Prior median lines of treatment = 3, CRS = 90% but mostly Grades 1 or 2. CR=74% (88% ORR) and MRD- = 93% (10-5). Median PFS = 20 months (CR patients, mPFS = 28 months). Med OS = 36 months (CR patients, median overall survival = not reached). Presenter spoke of one of his patients with 5 prior lines at 39 month PFS. {Mon-579-BY Wang}
  2. Janssen reported on their early results of CARTITUDE-1, a study of JNJ-4528, a licensed product of Legend-2 (see above). So far for 29 patients with a median lines of treatment of 3, they have similar results to above: All but 2 patients had CRS occurring 2-12 days post-infusion. 100% of patients were MRD-, but at different rates ranging from 10-4 to 10-6. ORR was a rather astounding 100% (CR 69%) for target 75x106 dosage, so findings so far are consistent with Legend-2. Four patients had extramedullary disease and 2 of these patients were among the patients who relapsed, including one who died from grade 5 cytokione release syndrome. The results were so impressive that Dr. Madduri momentarily stopped her talk and asked the audience to look and think about the 100% response graph she was presenting. {Mon-577-D Madduri}
  3. Another China study for N=29 pt, prior median lines of treatment = 3 examined results for sequential CD-19 and BCMA CAR-T It showed 85% ORR (although 3 relapsed), mPFS 16 months, 2yr overall survival at 56%, and one-third patients MRD- at 10-6. {Mon-578-L Yan}
  4. And yet another study from China for CT103A presented results from a fully human BCMA CAR-T in 18 patients with more than 3 lines of treatment. Early data showed ORR=100% and CR/sCR=68%. One patient had this CAR-T after relapsing from a murine (mouse derived) CAR-T and is doing well. The goal of this “human” CAR-T is better safety while similar efficiency at a lower dosage, although all patients still experienced CRS, mostly G1-2. The first patient treated has experienced about 1 yr of cell persistence. {Mon-582-C Li}
  5. Since BCMA CAR-T’s have generally had short PFS, another group from China created a “bi-specific” CAR-T, targeting both BCMA and CD38 (the latter being the same cancer cell surface antigen targeted by Dara), referred to as BM38 CAR-T cells. The abstract reported that for N=16, 14 achieved ORR and 8 (50%) reached sCR. CRS was seen in 14 of 16 patients but only 4 of those >= Grade 3 and these were resolved with tocilizumab. {Mon-930-H Mei}
  6. I didn’t attend a talk to update the results of bb21217 enriched for memory T cells, specifically designed to sustain CAR-T cell persistence with N=38 ORR = 83%. {Mon-927-J Berdeja}

OTHER RESULTS

  1. This study examined outcomes in MM based on comorbidities and race. It noted that Blacks are less likely to have an SCT than Whites; however with treatment access at the VA, they actually do better (hazard ratio=.79).  And on average, Blacks are dx’d 3 yrs younger than Whites (66 vs 69). It’s also important to note that clinicians might misperceive kidney involvement due to higher reference creatinine levels for Blacks, which could mean holding back treatment with Rev, which is excreted though the kidneys. This highlights the importance of including all races in clinical trials. {Sun-383-M Schoen}
  2. This study showed that there was no difference in overall survival between Medicare and private insurance for myeloma patients getting transplants. {Sun-424-G Ravi}
  3. This study showed the importance of having both MRD- and PET-CT- rather than only one. In the Cassiopeia study (VTd +/- Dara), “double negativity” confirmed better PFS in both arms as well as better PFS in the Dara over non-Dara arm. Longer follow-up may result in both methods being used as a predictive surrogate marker. {Mon-692-P Moreau}
  4. Primary Plasma Cell Leukemia is a very serious disease and I don’t remember seeing trials for either this or secondary PCL because it’s also rare. This Ph 2 study (N=21) showed that KRd can induce deep responses after 4 cycles with ORR 93% (CR=33%, VGPR= 47%). However 14 of 21 patients experienced serious adverse events ( toxicity G3=20% and G4= 27%) during the first 6 months, many during the first cycle but these were managed. Note, if a patient comes in with CNS involvement, Pom which crosses the blood brain barrier, should be considered instead of Rev. {Mon-693-N Van De Donk}
  1. This study provided a progression free survival analysis of the bone strengtheners Denosumab (Xgeva) vs Zoledronic Acid (Zometa) for 930 myeloma patients planning to get an SCT. Previously Denosumab showed a median PFS benefits of 10.7 months and this study showed a similar median PFS benefit of 10.4 months (46.1 vs 35.7 months) for patients intending to get an SCT. While this favors Denosumab, one needs to consider the “rebound effect” after stopping Denosuma – the increased risk of osteoporosis, so some recommend using Zometa to prevent that. Plus there’s the higher cost of Denosumab. {Mon-606-E Terpos}

SUMMARY

This year’s ASH continued to amaze me with so many studies in Myeloma, focusing on all stages from Smoldering Myeloma to myeloma Induction through Relapse. While some years at ASH are focused on the usage of newly approved drugs, this past year only had one approval - Selinexor for a limited audience. Rather, the importance of this year’s ASH is that we have another year of results in different area of myeloma treatment from High Risk smoldering myeloma patients to Induction therapies for newly diagnosed, to Immune Therapies initially being tested for relapsed/refractory myeloma patients. For High Risk SMM patients we’ve seen results of Rev or Rev-dex to delay progression and are now starting to see early outcomes for “curative” intense treatments.  For induction, we’re seeing more and more combinations including Dara, which results in better responses. It seems we’re not far away when 4 drugs become the standard initial regimen.

And for Relapsed/Refractory myeloma patients, we have more trial readouts for Immune Therapies such as CAR-T, ADC (Antibody Drug Conjugates), Bi-Specific T-Cell Engagers (more focus on AMG-701 with easier administration) and naked mAb’s (TAK-079, Isatuximab).  Look at all the new drugs and CAR-T trials with another year’s worth of updates, especially the N (number of patients involved).  Results are early and these trials need larger patient numbers, better understanding of dosages, longer follow-up, and perhaps better prognosis assessment tools (see CAR-T).  While we patients are anxious for approval of this treatment, I think back to SCT’s for Myeloma which were first tried in 1984 but not approved until the early 1990’s. However, there continues to be an incredible interest by researcher and clinicians to find better Myeloma treatments, and it is likely that CAR-T treatment will progress just as SCT progressed.  Soon we’ll be learning if earlier treatment in the smoldering myeloma stage can delay progression to myeloma and even a possible cure for some.  And soon MRD, an excellent prognostic measurement, will hopefully be used to help guide treatment decisions.

For someone diagnosed with stage III multiple myeloma 25 years ago with only 2 treatment options available (MP or VAD-SCT) and given 2-3 years expected survival, I’ve seen incredible progress since 2003 when Velcade was first approved. While there continues to be unanswered questions, we now have many more effective treatments for myeloma, providing patients with better opportunities to manage their disease.

 GLOSSARY (according to Jack)

Drug (brand names) by Drug Class/Category

IMID – Immunomodulary Drug

T – Thalidomide

R – (Lenalidomide) Revlimid

Pom – Pomalidomide (Pomalyst)

 

PI – Proteasome Inhibitor

V- Velcade (Bortezomib)

Cfz, K – Carfilzomib (Kyprolis)

I, Ixa – Ixazomib (Ninlaro)

 

mAb – Monocloncal Antibody

D, Dara – Daratumumab (Darzalex)

E, Elo – Elotuzumab (Empliciti)

Isa – Isatuximab (SAR650984)

 

HDAC -  histone deacetylase inhibitors

Pano – Panobinostat (Farydak)

 

Nuclear Export Inhibitor

S, Sel- Selinexor (Xpovio)

 

Steroids

P – Prednisone

D or d - Dexamethasone

 

Chemotherapy Drugs

C – Cyclophosphamide (Cytoxan)

M – Melphalan

 

Treatment Measurements

EFS – Event-free Survival

ORR – Overall response (>=PR)

OS –  Overall Survival

PD – Progressive Disease

PFS – Progression-free Survival

PFS2 – PFS + next-line treatment PFS

TTP - Time to Progression

TTR - Time to Respond

Treatment Response

CR – Complete Response: No sign of MM (0 M-spike)

nCR – Near CR (positive M-spike, may be same as VGPR)

MR – Marginal Response: 0-50% reduction in MM

PR- Partial Response: 50% reduction in MM

SD – Stable Disease i.e. no response but also not worse

sCR-Stringent CR: CR+ normal FLC & no clonal cells

VGPR – 90% reduction in MM

MRD – Minimum Residual Disease typically by Flow Cytometry (NGF) or DNA sequencing (NGS) to provide more sensitive measure of MM (e.g. 10-5 or 10-6)

 

Side Effects

AE (ASE) – Adverse Event (Adverse Side Effects)

DVT - Deep Vein Thrombosis (blood clots)

MTD – Maximum Tolerated Dose

ONJ – Osteonecrosis of the Jaw

PE – Pulmonary Embolism

PN – Peripheral Neuropathy

QOL – Quality Of Life

VTE - Venous Thromboembolism (PE + DVT)

CRS – Cytokine Release Syndrome

 

Tests/When to treat?/Other

CRAB – High Calcium, Renal, Anemia, and Bone…

CRABi – CRAB + “i” increased infections

FLC – Free Light Chain

 

SCT – Auto stem cell transplant.

TE, NTE – Transplant Eligible of Not TE

 

LOT – Lines of Therapy

 

ND – Newly Diagnosed

RR – Relapsed/Refractory

 

ADC -  Antibody Drug Conjugate

BiTE – Bi-specific T-Cell Engager

“d” and “D” – Typically both mean Low-dose Dex (40 mg/week) these days

MGUS – Monoclonal Gammopathy of Undetermined Significance

SMM – Smoldering MM

Pt(s) – Patient(s)

n - Number of pts

R/R- Relapsed/Refractory, Ref defined progressing while on Tx or within 60 days.

HR – High Risk

 

 

 

 
Jenny A
About the Author

Jenny A - Myeloma survivor, patient advocate, wife, mom of 6. Believer that patients can help accelerate a cure by weighing in and participating in clinical trials. Founder of Myeloma Crowd, Myeloma Crowd Radio and the CrowdCare Foundation.

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