| Objective:To explore the clinical curative effects of low dose arsenous acid/retinoid on myelodysplastic syndrome(MDS)of children,on different subtypes and risk degrees of MDS.Method:14 cases(male 6 cases,female 8 cases,median age 9 years,median disease duration 17 months)were enrolled in this study,where 6 cases(median disease duration 47.5 months)were not initial diagnostic patients who ever applied CsA or other drugs for a long period or discontinuously.Diagnostic classification and prognostic grading are based on Blood Disease Diagnosis and Curative Effect Standard(third edition,Zhang Zhinan),judgement on MDS Classification Standard issued by WHO in 2008 and Revised International Prognostic Scoring System(IPSS-R)for Myelodysplastic Syndrome in 2012.We choose the regime of induction of low dose of ATO/ATRA for the 14 cases,of whom treated for more than one course were measurable cases.Curative effect evaluation is based on the criteria of MDS curative effects of MDS International Working Group(IWG).Results:1.①In the 14 patients,clinical manifestations for anemia(<99g/L),thrombocytopenia(<99×109/L)and neutropenia(<0.8×109/L)are 12 cases(12/14),12 cases(12/14)and 4 cases(4/14),respectively.Meanwhile,patients of pancytopenia and marrow blasts less than 5%are 10 cases(10/14)and 11 cases(11/14),respectively.The decreased degree of each lineage blood cells,the appearing frequency,cases of pancytopenia,cases of marrow blasts less than 5%are not related to whether it is the initial diagnosis(P>0.05).②In the test of karyotypes,there are 8/14 cases(with good,middle and poor karyotypes 7cases,3cases and 4 cases respectively)with karyotype changes.There are 6 cases(6/8)with good karyotypes(normal karyotypes 5 cases and with 5q-1 case)in the newly-diagnosed group,while 1 case(1/6)in the non-newly-diagnosed group,and there are obvious differences of good karyotypes in the two groups(P<0.05).2.Clinical efficacy of 14 cases:①There are 6 cases of CR,2 cases of mCR and HI,2 cases of HI,2 cases of SD,and 2 cases of PD according to the short-term clinical curative effects.The short-term effective rate is 85.71%(12/14),and the clinical efficacy of newly-diagnosed group is obviously better than the non-newly-diagnosed group(P<0.05);It the 12 patients of having acquired clinical curative effect,cases of having acquired effective evaluation account for 57.14%(8/12),and the median time of acquiring clinical curative effect is ltreatment course(1～5),short-term clinical efficacy of 11 cases of RCC is 81.82%(9/11);and there are no significant differences between the medium and low risk group and the high risk group and above it(P>0.05).②To follow-up time,medium PFS of the 14 children is 18 months(8～56);while medium OS is 27.5 months(10～73).We have not found the regime can change the abnormal karyotypes of MDS patients.3.In the process of treatment,no obvious adverse reaction was found in all cases,and 10(10/14)children are still in the maintenance treatment.Conclusions:1.The mechanism of the regime treating medullary poison lowe is detoxification and eliminating pathogens.Eliminating toxic pathogens,and vital-qi recovery;2.In order to reduce the missed diagnosis rate of MDS,FISH test should be used routinely to look for cytogenetic evidence;3.The long course of the disease and poor karyotypes may play an important role in the short-trerm curative efficacy of the therapeutic regime;4.The favourable short-term clinical curative effect and safety have improved the quality of patients’ life. |
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