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.
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
Olaf
O
Witt
Susanne
S
Hämmerling
Clemens
C
Stockklausner
Jens-Peter
JP
Schenk
Patrick
P
Günther
Wolfgang
W
Behnisch
Bajes
B
Hamad
Naima Ali
NA
Al Mulla
Andreas
A
Kulozik
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