Abstract
New treatments are urgently needed for stage IV NB, which becomes resistant to chemotherapy (CT) in 75% of cases. We have treated 6 children (ch) with refractory NB with HD L-PAM, either 120 mg/m2 (4 ch) or 180 mg/m2 (2 ch), in 3 divided doses, followed by reinfusion of bone marrow (BM) previously frozen in liquid N2 during BM remission. The ch were isolated during the neutropenic phase and supported with red cells and platelets, and hyperalimentation via Broviac catheters.Toxicity: all 6 recovered hemopoietic function, in median periods of 28 days (neutrophils 500), 34 days (retics 1%), and 35 days (platelets 50,000). Gastrointestinal side effects were severe but short-lived. 1 ch developed hepatic venocclusive disease, which regressed completely with supportive measures. 3 ch developed sepsis while neutropenic. 1 ch died after 3 mo complete response (CR) from complications of prior abdominal surgery and had no tumor at autopsy.Responses: 120 mg/m2 (4 ch)--1 CR (6 mo), 1 PR (1 mo), 2 NR. 180 mg/m2 (2 ch)--2 CR (3 mo and 4+ mo).Conclusions: 1. HD L-PAM is clearly active against NB. 2. Autologous BM reinfusions protect against severe myelosuppression. 3. BM autografting may uncover other dose-limiting toxicities of L-PAM. 4. L-PAM should be combined with other agents in further phase II and III studies.
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