13-cis retinoic acid treatment of a patient with chemotherapy refractory nephroblastomatosis.
A 9-month-old girl presented with massive bilateral diffuse nephroblastomatosis. After response to actinomycin D and vincristine over a period of 1 year, the nephroblastomatosis continuously progressed under this treatment. As retinoic acid signaling is critical for normal renal development and nephroblastomatosis seems histologically as undifferentiated embryonal tissue, we added 13-cis retinoic acid to the chemotherapy regimen. Three months thereafter, kidney volumes declined significantly over a period of 1 year. Interestingly, nephroblastomatosis-associated acquired von Willebrand disease also resolved. Retinoic acid maybe a novel nontoxic treatment option for nephroblastomatosis requiring further systematic evaluation.
Witt
Olaf
O
CCU Pediatric Oncology, German Cancer Research Center, Department of Pediatric Oncology, Hematology, Immunology and Pneumonology, Children's Hospital, University of Heidelberg, Heidelberg, Germany. o.witt@dkfz.de
Hämmerling
Susanne
S
Stockklausner
Clemens
C
Schenk
Jens-Peter
JP
Günther
Patrick
P
Behnisch
Wolfgang
W
Hamad
Bajes
B
Al Mulla
Naima Ali
NA
Kulozik
Andreas
A
D000903:Antibiotics, Antineoplastic / Q000008:administration & dosage; D000972:Antineoplastic Agents, Phytogenic / Q000008:administration & dosage; D003609:Dactinomycin / Q000008:administration & dosage; D003879:Dermatologic Agents / Q000008:administration & dosage; D019008:Drug Resistance, Neoplasm; D005260:Female; D006801:Humans; D007223:Infant; D015474:Isotretinoin / Q000008:administration & dosage*; D007668:Kidney / Q000473:pathology; D007680:Kidney Neoplasms / Q000188:drug therapy* / Q000473:pathology; D008279:Magnetic Resonance Imaging; D011230:Precancerous Conditions / Q000188:drug therapy* / Q000473:pathology; D014750:Vincristine / Q000008:administration & dosage
D000903:Antibiotics, Antineoplastic; D000972:Antineoplastic Agents, Phytogenic; D003879:Dermatologic Agents; D003609:Dactinomycin; D014750:Vincristine; D015474:Isotretinoin