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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