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@ARTICLE{Mrdter:306679,
author = {T. E. Mürdter and W. Schroth and M. P. Goetz and R.
Tremmel and S. Igel and E. Schaeffeler and S. Jäger and S.
Loibl and A. Gerteis and L. Pfaff and C. Bechtner and D.
Wrobel and I. Bernhöft and I. Fischer and C. Meisner and M.
Block and H. Brauch$^*$ and M. Schwab$^*$},
collaboration = {G. T. C. Group},
othercontributors = {C. Bechtner and I. Bernhöft and V. Bjelic-Radisic and M.
Burkhardt and M. Darsow and G. Deutsch and T. Dewitz and W.
Dietz and M. Eichbaum and E. Faust and T. Göhler and S.-T.
Graßhoff and J. Hackmann and C. Hänle and A. Hartkopf and
B. Heinrich and C. Kahl and G. Kaltenecker and A.
Kleine-Tebbe and H.-C. Kolberg and T. Krauß and T. Kühn
and G. Kunz and A. Ligl-Löhner and M. Negwer and U. Rhein
and G. Rogmans and J. Rom and S. Schmatloch and M.
Schwab$^*$ and G. Stalzer and A. Stefek and H.-J.
Strittmatter and J. Terhaag and C. Uleer and K. Wimmer-Freys
and D. Wrobel and D.-M. Zahm},
title = {{S}upplementation of {T}amoxifen with {L}ow-{D}ose
{E}ndoxifen in {P}atients with {B}reast {C}ancer with
{I}mpaired {T}amoxifen {M}etabolism ({TAMENDOX}): {A}
{R}andomized {C}ontrolled {P}hase {I}/{II} {T}rial.},
journal = {Clinical cancer research},
volume = {31},
number = {23},
issn = {1078-0432},
address = {Philadelphia, Pa. [u.a.]},
publisher = {AACR},
reportid = {DKFZ-2025-02672},
pages = {4903 - 4911},
year = {2025},
abstract = {:Tamoxifen undergoes bioactivation to its active metabolite
(Z)-endoxifen, which blocks estrogen-dependent breast tumor
growth at high potency. We tested the feasibility and safety
of supplementing standard tamoxifen therapy with low-dose
(Z)-endoxifen in patients with breast cancer with
compromised tamoxifen bioactivation.:We conducted a
prospective, interventional, three group randomized trial
including 235 patients with hormone receptor-positive breast
cancer who received standard tamoxifen therapy (20 mg/day).
Patients were stratified by CYP2D6 genotype (n = 78),
defining poor, intermediate, and normal metabolizers, or by
baseline (Z)-endoxifen plasma concentration (n = 78),
defining ≤15, 15 to 25, and ≥25 nmol/L. Co-treatment
with (Z)-endoxifen 3 and 1.5 mg/day or placebo was
performed, respectively. A control group (n = 79) received
placebo regardless of metabolizer phenotype. The primary
endpoint was the number of patients with (Z)-endoxifen
levels >32 nmol/L after 6 weeks of treatment. Adverse events
were continuously monitored.A higher proportion of patients
in both intervention groups achieved target concentrations
>32 nmol/L compared with control (P < 0.0001). At 3 mg
(Z)-endoxifen supplementation, $92.3\%$ of CYP2D6 poor
metabolizer patients and all patients with baseline
(Z)-endoxifen ≤15 nmol/L achieved the target
concentration. At 1.5 mg (Z)-endoxifen supplementation,
$88\%$ of CYP2D6 intermediate metabolizer patients and
$95\%$ of patients with 15 to 25 nmol/L baseline
(Z)-endoxifen levels achieved the target concentration.
Similar proportions of patients receiving (Z)-endoxifen
(6/80, $7.5\%)$ or placebo (8/155, $5.2\%)$ experienced
grade 3 adverse events.Adding low-dose (Z)-endoxifen to
standard tamoxifen is safe and provides a new approach to
personalized antiestrogen treatment for patients with low
endoxifen plasma levels.},
keywords = {Humans / Female / Breast Neoplasms: drug therapy / Breast
Neoplasms: pathology / Breast Neoplasms: genetics / Breast
Neoplasms: metabolism / Tamoxifen: administration $\&$
dosage / Tamoxifen: analogs $\&$ derivatives / Tamoxifen:
pharmacokinetics / Tamoxifen: adverse effects / Middle Aged
/ Cytochrome P-450 CYP2D6: genetics / Aged / Adult /
Antineoplastic Agents, Hormonal: administration $\&$ dosage
/ Antineoplastic Agents, Hormonal: adverse effects /
Prospective Studies / Treatment Outcome / Aged, 80 and over
/ Genotype / Tamoxifen (NLM Chemicals) / Cytochrome P-450
CYP2D6 (NLM Chemicals) / 4-hydroxy-N-desmethyltamoxifen (NLM
Chemicals) / Antineoplastic Agents, Hormonal (NLM
Chemicals)},
cin = {TU01},
ddc = {610},
cid = {I:(DE-He78)TU01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41036998},
doi = {10.1158/1078-0432.CCR-25-2103},
url = {https://inrepo02.dkfz.de/record/306679},
}