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V. Jachiet et al.
Comparison with two prospective cohorts of patients with only immune thrombocytopenia or with only myelodysplastic syndrome/chronic myelomonocytic leukemia
Patients with MDS/CMML-associated ITP were matched for age (±5 years) and sex to patients with primary ITP included between 2013 and 2019 in the multicenter Cytopeń ies Auto- immunes: Registre Midi-Pyreń eÉ N (CARMEN) registry at a 1:2 ratio. CARMEN is a registry, established in June 2013, that follows prospectively incident ITP adult patients (≥18 years) in the Midi- Pyreń eé s region (southern France, 3 million inhabitants).13,14 The absence of myelodysplasia in all primary ITP controls in this study was confirmed by bone marrow examinations.
Patients with MDS/CMML-associated ITP were also matched for age (±5 years), sex, type of disorder (MDS or CMML) and IPSS- R score (≤ or >3.59) to patients with MDS/CMML without ITP beginning in 2003 in the multicenter Groupe Francophone des Myélodysplasies (GFM) registry of MDS/CMML at a 1:4 ratio.
Statistical analysis
Continuous variables are presented as the mean ± standard deviation or as the median (range), as appropriate. Qualitative variables are presented as a number (%). The Mann-Whitney test and t-test were used to compare continuous variables, and the χ2 and Fisher exact tests were used to compare qualitative variables. Overall survival was calculated from the date of MDS/CMML
diagnosis to death or the last date of follow-up. Leukemia-free sur- vival was calculated from the date of MDS/CMML diagnosis to the date of transformation to AML. Overall and leukemia-free sur- vival were analyzed with the log-rank test, and the results are expressed using Kaplan-Meier methods. A P-value <0.05 was con- sidered statistically significant. Statistical analyses were performed
Table 1. Baseline characteristics and outcome of patients with myelodysplastic syndrome/chronic myelomonocytic leukemia (MDS/CMML)-asso- ciated immune thrombocytopenia (ITP), MDS/CMML without ITP and primary ITP.
using GraphPad Prism 5.0 for Mac (GraphPad Software CA, USA).
Results
TM
, La Jolla,
Characteristics of patients with myelodysplastic syndrome/chronic myelomonocytic leukemia-associated immune thrombocytopenia
Between January 1999 and July 2019, we screened 77 patients with MDS/CMML and ITP and ultimately includ- ed 41 patients from 16 French hospitals (Figure 1). The median age at diagnosis was 77 years (range, 35-92) in the MDS/CMML group with ITP, and 41% of the patients were women (Table 1). The median IPSS-R score was 3 (range, 1-5), and 30 (73%) patients had low-risk MDS. ITP was diagnosed concomitantly with MDS/CMML (± 3 months) in 17 (41%) patients. The diagnosis of ITP pre- ceded that of MDS/CMML (-116 to -6 months) in 16 (39%) patients and occurred after the diagnosis of
Age at diagnosis, years
Female sex
ITP features
Platelet count at ITP diagnosis, x 109/L Khellaf bleeding score > 8
Severe bleeding
Antiplatelet antibodies
Direct antiglobulin test
Antinuclear antibodies
Polyclonal hypergammaglobulinemia
MDS/CMML features at inclusion MDS
CMML
Platelet count, x 109/L
Hemoglobin, g/dL
Neutrophils, x 109/L
Bone marrow blasts, %
Abnormal karyotype
Low-risk MDS/CMML
IPSS
IPSS-R
Secondary MDS
MDS treatment (except EPO and transfusion) Number of treatment lines for MDS
Outcome
Follow-up, months
AML transformation
Deaths
Deaths related to MDS/CMML or a specific therapy
MDS/CMML-ITP n=41
77 [35-92]
17 (41)
15 [1-90] 11/38 (29) 8/31 (26) 7/16 (44) 12/22 (55) 12/29 (41) 12/34 (35)
17 (41)
24 (59) 15 [1-90] 11.8 [5-15] 3.9 [1.2-23] 3 [0-13] 10/34 (29) 30/41 (73) 0.5 [0-2] 3 [1-5] 4/40 (10) 9/41 (22) 1 [1-2]
66 [1-176] 4/41 (10) 17/41 (41) 13/17 (76)
Primary ITP
n=75
N (%) or mediian [range]
76 [35-92]
29 (39)
11 [1-91] 21/75 (28) 3/75 (4) -
- 26/57 (46) 9/51 (18)
11 [1-91] -
-
-
-
-
-
-
-
-
-
- - - -
MDS/CMML without ITP P n=200
78 [28-92] 0.33
75 (38) 0.89
137 [8-1488]
- 0.92
-
- - - - - 0.71 - 0.07
84 (42) 0.95 116 (58)
3 [0-29] 0.26 63/197 (32) 0.73 155/200 (78) 0.55 0.5 [0-3.5] 0.66 2.5 [0-8] 0.39 14/97 (14) 0.14 45/200 (23) 0.94 1 [1-3] 0.91
43/200 (22) 0.01†
18/43 (42) 0.02†
<0.01‡
0.01*
137 [8-1488]
10.9 [6-16] 0.14 3.9 [0.3-68] 0.72
<0.01‡
23 [0-106]
15/200 (8) 0.63
<0.01†
ITP: immune thrombocytopenia; MDS: myelodysplastic syndrome; CMML: chronic myelomonocytic leukemia; IPSS: International Prognostic Scoring System; IPSS-R: Revised International Prognostic Scoring System; EPO: erythropoietin; AML: acute myeloid leukemia. *P<0.05: MDS/CMML-associated ITP versus primary ITP; †P<0.05: MDS/CMML-associ- ated ITP versus MDS/CMML without ITP; ‡P<0.05: MDS/CMML-associated ITP versus primary ITP versus MDS/CMML without ITP.
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