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Efficacy and toxicity of a paediatric protocol in teenagers and young adults with Philadelphia chromosome negative acute lymphoblastic leukaemia: results from UKALL 2003
Author(s) -
Hough Rachael,
Rowntree Clare,
Goulden Nick,
Mitchell Chris,
Moorman Anthony,
Wade Rachel,
Vora Ajay
Publication year - 2016
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.13847
Subject(s) - medicine , pediatrics , minimal residual disease , confidence interval , toxicity , acute lymphocytic leukemia , adverse effect , chemotherapy , young adult , prospective cohort study , lymphoblastic leukemia , leukemia
Summary Despite the substantial outcome improvements achieved in paediatric acute lymphoblastic leukaemia ( ALL ), survival in teenage and young adult ( TYA ) patients has remained inferior. We report the treatment outcomes and toxicity profiles observed in TYA patients treated on the UK paediatric ALL trial, UKALL 2003. UKALL 2003 was a multi‐centre, prospective, randomized phase III trial, investigating treatment intensification or de‐escalation according to minimal residual disease ( MRD ) kinetics at the end of induction. Of 3126 patients recruited to UKALL 2003, 229 (7·3%) were aged 16–24 years. These patients were significantly more likely to have high risk MRD compared to 10–15 year olds (47·9% vs. 36·6%, P = 0·004). Nonetheless, 5‐year event‐free survival for the TYA cohort (aged 16–24 years) was 72·3% [95% confidence interval ( CI ): 66·2–78·4] overall and 92·6% (95% CI : 85·5–99·7) for MRD low risk patients. The risk of serious adverse events was higher in patients aged ≥10 years compared to those aged 9 or younger ( P < 0·0001) and novel age‐specific patterns of treatment‐related toxicity were observed. TYA patients obtain excellent outcomes with a risk‐ and response‐adapted paediatric chemotherapy protocol. Whilst those aged 10 years and older have excess toxicity compared with younger patients, the age association is specific to individual toxicities.