Treatment of Myeloma Bone Disease
James R. Berenson, MD
Medical & Scientific Director
Institute for Bone Cancer & Myeloma Research
West Hollywood, CA

Clinical Consequences of
Myeloma Bone Disease
· Pathological fractures
­ Non-vertebral
­ Vertebral compression
· Spinal cord compression/collapse
· Radiation therapy
· Surgery to bone
· Hypercalcemia
· Bone pain
· Use of analgesics
· Quality-of-life effects
· Survival

Prevalence of Skeletal Complications in
21 months among MM Patients
Total
Pathologic
fracture
Radiation to
bone
Hypercalcemia of
malignancy
Surgery to bone

Spinal cord

compression
0
10
2030
40
5060
Patients With SREs, %
9-month data.
Placebo arm of pamidronate randomized trial.
Berenson JR et al. N Engl J Med. 1996;334:488-493.
Berenson JR, et al. J Clin Oncol. 1998;16:593-602.

Clinical Consequences of
Myeloma Bone Disease
· Pathological fractures
­ Non-vertebral
­ Vertebral compression
· Spinal cord compression/collapse
· Radiation therapy
· Surgery to bone
· Hypercalcemia
· Bone pain
· Use of analgesics
· Quality-of-life effects
· Survival

Kyphoplasty: A Minimally Invasive
Fracture Reduction Procedure
KyphX Introducer Tool
KyphX IBT inflation:
KyphX IBT Removal:
Kit:
· Reduces the fracture.
· Leaves a defined cavity
· Allows precise,
· Compacts the bone.
and trabecular dam that
minimally invasive access
· May elevate
can be filled with an
to the vertebral body.
endplates.
approved bone void filler
· Provides working
of the physician's
channel.
choice.

Kyphoplasty: Use in Myeloma Patients w/
Vertebral Compression Fractures
· 55 procedures in 18 patients
· Assess safety and efficacy using the Short Form
Health Survey (SFHS) scoring system at
pre, 1, 6, 12, 36, & 52 weeks
­ Body pain, Physical function, Vitality, Social Function
· Results
­ Levels: T6-L5 (majority (56%) from T11-L5)
­ No major complications
­ Improvement in SFHS assessments
Dudeney et al. J Clin Oncol 2002

The CAFÉ Study
A Multicenter, Prospective,
Randomized, Controlled Study to
Compare Balloon Kyphoplasty to
Non-Surgical Fracture Management*
in the Treatment of Cancer Patients
with Painful Vertebral Body
Compression Fractures
*Allowed to have kyphoplasty 30 days later

Bisphosphonates in the Treatment of
Myeloma Bone Disease: Randomized Studies
· Oral
­ Etidronate* - Belch et al, J Clin Oncol 1991
­ Clodronate* - Lahtinen et al, Lancet 1992;
McCloskey et al, Br J Haematol 1998
­ Pamidronate* ­ Brincker et al, Br J Haematol 1998
· Intravenous
­ Pamidronate* ­ Berenson et al, N Engl J Med 1996
­ Ibandronate* ­ Menssen et al J Clin Oncol 2002
­ Zoledronic acid+ ­ Berenson et al, Cancer 2001;
Rosen et al, The Cancer J 2001
* Placebo-controlled;
+ Pamidronate-controlled

Effect of Monthly Intravenous Pamidronate (90 mg) in
Reducing Skeletal Events in Patients with Advanced
Multiple Myeloma: A Phase III Trial
Berenson JR, et al. N Engl J Med. 1996;334(8):488-493.

OH
N
O
Zoledronic acid
N
P
O
OH
P
OH
· Zoledronic acid belongs to a
HO
OH
new class of bisphosphonates
1,2
· Heterocyclic, nitrogen-containing
bisphosphonate composed of:
­ A core bisphosphonate moiety
­ An imidazole-ring side chain containing 2 critically
positioned
nitrogen atoms
· 2-3 logs more potent than pamidronate
in preclinical studies
1,2
1. Green J, et al. J Bone Miner Res. 1994.
2. Green J, et al. Pharmacol Toxicol. 1997.

Zoledronic Acid in Myeloma and
Breast Cancer Patients:
Protocol 010 Trial Design
· 24-mo dosing regimen
­ Pamidronate 90 mg every 3 to 4 wk/
120-min infusion
­ Zoledronic acid 4 mg and 8/4 mg every 3 to
4 wk/
5-min amended to 15-min infusion
­ Double-blind, double-dummy
· Study duration: 25 mo
· Patients received oral vitamin D 400 IU
and calcium 500 mg

Breast Cancer and Multiple Myeloma
Efficacy Summary
Time to
Multiple-
Proportion
first SRE
Mean skeletal
event analysis
with SRE, %
(median)*
morbidity rate*
hazard ratio*
ZA** 4 mg
47
376
1.04
0.841
Pam 90 mg
51
356
1.39
--
P value
.243
.151
.084
.030
*Hypercalcemia of malignancy is included as a skeletal-related event
** ZA- zoledronic acid
Extension data

Multiple Myeloma
Time to First Serum Creatinine Increase*
100
%
80
crease, 60
ntiu 40
o
ith
n
Hazard ratio P value
20
w
ZOMETA
® 4 mg­15 min
91
0.764
.502
ts
Pamidronate 90 mg-2 hr 84
0
tiena
0
120
240
360
480
600
720
P
Time, days
ZOMETA 4 mg 91
76
71
57
32
30
18
Pamidronate
84
71
62
50
23
17
8
*Post­15-minute infusion amendment.
Versus pamidronate infused over 2 hours
After start of study drug.

How to Approach the Patient w/ Rises in
Creatinine While on a Bisphosphonate (BP)
· Look for possible causes besides the BP
­ The Cancer (myeloma, etc)
­ Other co-morbid diseases (diabetes, hypertension, etc)
­ Medications (NSAIDS, COX-2 inhibitors, statins, etc)
­ Consider a kidney biopsy
· If the BP still may be the cause, hold the dose until the
creatinine returns to baseline, & since the rate of infusion
relates to kidney toxicity, SLOW the infusion down
­ To 60 min for Zometa
­ To > 4 hours for Aredia
· If problems persists, consider switching to the other IV BP
· If does not improve the problem, discontinue intravenously
administered BPs and consider Fosamax orally

Osteonecrosis of the Jaw (ONJ) &
Bisphosphonate (BP) Use
· Observed among CA patients receiving IV BPs
(both pamidronate & zoledronic acid)
· Less frequent among pts receiving oral BPs
· Relationship to BPs unproven
· Treatment
­ Good oral hygiene
­ Peridex (chlorhexidine gluconate) rinse or Arestin
(minocycline hydrochloride) periodontal pockets
­ Intermittent antibiotics, antifungals or antivirals
­ Keep surgery to a minimum
­ No evidence that discontinuation of BP changes
course of jaw problem
· Prophylaxis- Excellent oral hygiene

ONJ in Myeloma Patients-
The IMBCR Experience
·
6 cases of ONJ
·
Range of severity
­ 3 patients required intermittent antibiotics (Aredia + Zometa,
Zometa only in 2) - remain on bisphosphonate treatment
­ 1 patient recently diagnosed with minor temporary discomfort
(Aredia only) - remains on treatment
· Largely resolved with clarithromycin PO
­ 2 patients (Aredia + Zometa, Zometa only) discontinued
bisphosphonate secondary to significant effect on mastication
·
Status of myeloma
­ 3 in long-term complete remission (auto-PSCT, VAD, thalidomide)
­ 1 in near complete remission (on steroids)
­ 2 with long-term indolent myeloma requiring
no other therapy
· 1 patient with 40% reduction in M-protein for > 4 yr

IV Bisphosphonates for
Patients With Myeloma Bone
Disease--Benefits vs Risks
Benefits
Risks
Fractures
ONJ ?
Radiotherapy
Renal
Bone pain
(infrequent)
Humeral fracture in
a myeloma patient

Monoclonal Gammopathy of
Undetermined Significance (MGUS)
· Pts w/ monoclonal immunoglobulin
· 3% of individuals over 70 years
· None of the clinical manifestations of
myeloma, however
· Bone effects
­ Increased osteoclastic activity
­ High rate of bone loss
­ Enhanced risk of fracture especially vertebral
compression fracture*
*Melton et al. J Bone Min Res 2004

Phase I/II Study of ZOMETA
® in MGUS
Patients with MGUS and osteopenia/osteoporosis (n=80)
(t-score < ­1)
ZOMETA 4 mg via 15-minute infusion
at 0, 6 and 12 months
Primary endpoint:
Secondary endpoints:
Posterior-anterior spine
Nondominant proximal femur BMD
BMD (0 & 13 mo)
Skeletal radiographs
in M-protein
Progression

Myeloma Bone Disease
Conclusions (I)
· Major clinical manifestation of MM
­ High risk of pathological fractures
­ Many patients receive radiotherapy
· Keep radiotherapy to a minimum
­ Side effects
­ Compromises bone marrow function
· Increasing recognition of the role of
kyphoplasty to treat vertebral
compression fractures

Myeloma Bone Disease
Conclusions (II)
IV zoledronic acid and pamidronate
­ Reduce skeletal complications
· Pathologic fractures
· Radiation therapy
­ Monitoring of kidney function
(assess monthly serum creatinine) necessary
although other causes more likely to cause
renal dysfunction
­ Osteonecrosis of the jaw may be associated
with bisphosphonate use
· Good oral hygiene important to emphasize
· Avoid invasive dental procedures
­ Potential anti-tumor effects being explored