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Table 2 Treatment and outcomes

From: Treatment of gastrointestinal stromal tumours in paediatric and young adult patients with sunitinib: a multicentre case series

  Imatinib response/dose/treatment duration Imatinib PFS and TTP Sunitinib response/dose/treatment duration/BSA Sunitinib PFS and TTP Overall follow up Outcome Adverse event/Toxicity during sunitinib treatment
Patient A SD then PD; 400 mg OD then 300 mg OD; 8mo PFS 17mo
TTP 17mo
SD liver metastasis; 50 mg OD 4/2 schedule, then 37.5 mg OD; 73mo; 1.52m2 PFS 73mo
TTP No progression
163mo AWD on sunitinib since 2008 Hand and foot syndrome /TG 1
Arterial hypertension/TG 1
Skin rash/TG 2
Anaemia/TG 2
Diarrhoea/TG 2
Hypothyroidism/TG 2
Patient B SD then PD;
400 mg OD; 9mo
PFS 8mo
TTP 8mo
SD then PD; 37.5 mg OD then 50 mg OD 4/2 schedule; 7mo; 1.39m2 PFS 6mo
TTP 6mo
159mo AWD on regorafenib Hypothyroidism/ TG 1
Diarrhoea/ TG 2
Skin rash/ TG 1
Patient C SD then PD; 400 mg OD then 200 mg OD; 9mo PFS 8mo
TTP 8mo
SD then PD; 25 mg OD then 37.5 mg OD then 25 mg OD; 23 months; 1.28m2 PFS 6mo
TTP 6mo
88mo AWD on imatinib/doxorubicin Cholecystitis /TG 3
Diarrhoea/TG 1
Patient D PD; 400 mg OD; 12mo PFS 12mo
TTP 12mo
SD then PD; 50 mg OD 4/2 schedule then 37.5 mg OD; 24mo; 1.39m2 PFS 23mo
TTP 23mo
260mo AWD on treatment (nilotinib, then imatinib + doxorubicin, then imatinib 800 mg OD Hypothyroidism/TG 3
Pyrosis/TG 1
Anaemia/TG 3
Thrombocytopenia/TG 2
Fatigue/TG 2
Amenorrhoea/TG 2
Discoloration of skin and hair/TG 2
Hand and foot syndrome/TG 2
Patient E SD; 400 mg BID then 200 mg OD then 300 mg OD; 7mo PFS 7mo
TTP NA – intolerance (neutropenia, fatigue, joint pain)
PD; 25 mg OD 4/2 schedule (reduced due to prior intolerance of imatinib); 5mo; 1.51m2 PFS 5mo
TTP 5mo
139mo AWD, PD on nilotinib after sunitinib. Slow PD- asymptomatic, no treatment since 2011 Abdominal pain/TG 2
Bone pain/TG 2
Fatigue/TG 2
Patient elected to discontinue sunitinib due to above side effects, PD then documented
Patient F NA NA PD; 50 mg OD 4/2 schedule; 1mo; 1.50m2 PFS 1mo
TTP 1mo
25mo DOD, after sunitinib also failed trametinib, regorafenib, a phase 1 clinical trial and pazopanib Fatigue/TG 1
Thrombocytopenia/TG 1
Patient G SD then PD; 400 mg OD; 14mo PFS 14mo
TTP 14mo
SD then PD; 37.5 mg OD then discontinued for 3mo for toxicity then restarted 12.5 mg OD increasing to 25 mg OD; 17mo; 1.65m2 PFS 17mo
TTP 17mo
76mo AWD, no treatment since sunitinib and subsequent SD under observation from 2012 Fatigue/TG 1
Oedema/TG 2
Epistaxis/TG 1
Headaches/TG 1
Patient H SD then PD; 400 mg OD then 800 mg OD: 49mo PFS 9mo
TTP 9mo
SD for duration but initial minor response; Initial dose 50 mg OD, lowest dose 12.5 mg OD; 17mo; 1.60m2 PFS 5mo
TTP 5mo
173mo AWD on imatinib Fatigue/TG 3
Mucositis/TG 3
Diarrhoea/TG 3
Patient I SD then PD; 400 mg OD; 1mo PFS 2mo
TTP 2mo
SD then PD then SDa; 50 mg OD then 37.5 mg OD; >42mo (RFA during treatment for liver metastases); b1.68m2 PFS 28mo
TTP 28mo
86mo AWD on sunitinib Mucositis /TG 2
Diarrhoea/TG 2
Hypertension/TG 2
  1. aPD liver lesion treated with RFA; bLast recorded July 2012
  2. AWD = alive with disease; BSA = Body surface area at start of sunitinib treatment (a, last record; b, during therapy); DOD = dead of disease; OD = once daily; mo = months; NA = not applicable; PD = progressive disease; PFS = progression-free survival; RFA = Radiofrequency Ablation; SD = stable disease; TG = toxicity grade; TTP = time to progression; 4/2 schedule = 4 weeks of daily treatment and 2 weeks no treatment