Analysis of efficacy and prognostic factors of lenalidomide treatment as part of a Dutch compassionate use program.
METHODS
To obtain efficacy and safety data on lenalidomide treatment outside of clinical trials, we analyzed the clinical data of 114 patients with refractory or relapsed multiple myeloma treated with lenalidomide on a compassionate use basis. The recommended treatment consisted of lenalidomide 25 mg given on days 1-21 of a 28-day cycle, in combination with dexamethasone. A median of 3 previous lines of therapy were given, including thalidomide in 91%. Most patients were treated until progression or intolerable toxicity.


RESULTS
The median number of cycles was 7 (range, 1-21+ cycles) with a maximum response after a median of 3 cycles (range, 1-10 cycles). The overall response rate was 69%, including complete response in 6%, very good partial response in 19%, and partial response in 44%. The response rate was not influenced by previous thalidomide and/or bortezomib treatment. The median time to progression (TTP) was 9 months and the median overall survival (OS) was 22 months. A significantly longer TTP was observed in patients who previously underwent allogeneic stem cell transplantation (12.5 months vs. 8 months; P = .036). Overall survival was significantly affected by performance status (P < .0001). Lenalidomide toxicity was predominantly hematologic (37%; Common Toxicity Criteria > or = 3) and the incidence of venous thrombotic events was low (5%) using the recommended prophylaxis.


CONCLUSIONS
This analysis confirms that, outside clinical prospective trials, treatment with lenalidomide is highly effective and feasible in heavily pretreated patients with multiple myeloma.
Kneppers
Evelien
E
Department of Haematology, University Medical Center, Utrecht, The Netherlands.
Lokhorst
Henk M
HM
Eeltink
Corien M
CM
Huls
Gerwin
G
Kersten
Marie José
MJ
Koedam
Jan
J
Minnema
Monique C
MC
van Oers
Marinus H J
MH
Raymakers
Reinier A P
RA
Schaafsma
Martijn R
MR
Vellenga
Edo
E
Wijermans
Pierre W
PW
Wittebol
Shulamiet
S
Sonneveld
Pieter
P
Zweegman
Sonja
S
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001897:Boronic Acids; D000069286:Bortezomib; D002986:Clinical Trials as Topic; D057176:Compassionate Use Trials; D003907:Dexamethasone / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D018450:Disease Progression; D005260:Female; D006801:Humans; D000077269:Lenalidomide; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy* / Q000209:etiology / Q000401:mortality; D011719:Pyrazines; D012008:Recurrence; D012106:Research; D033581:Stem Cell Transplantation; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives / Q000627:therapeutic use*
D001897:Boronic Acids; D011719:Pyrazines; D013792:Thalidomide; D000069286:Bortezomib; D003907:Dexamethasone; D000077269:Lenalidomide