Presentation Of Chronic Myeloid Leukemia Research Articles

A chronic myeloid leukemia presenting as panuveitis combined with retinal and choroidal vascular occlusion: a case report

BackgroundChronic myeloid leukemia (CML) can manifest ocular complications stemming from hematologic irregularities or direct infiltration of neoplastic cells. This article details the case of a patient with newly diagnosed CML exhibiting elevated platelet counts (PLT) who developed panuveitis accompanied by retinal vascular occlusion.Case presentationA 52-year-old woman experienced a notable decline in vision in her left eye over a 2-week period. Classical anterior uveitis, vitreous cavity opacity, optic nerve edema, and retinal vascular obstruction were observed. The right eye exhibited papilledema and retinal vein tortuosity. Despite admission, the condition of both eyes deteriorated, accompanied by a continuous increase in PLT. She was diagnosed with CML based on bone marrow biopsy and chromosomal examination. Following platelet apheresis therapy and chemotherapy, the condition of her right eye significantly improved, but the left eye’s condition remained irreversible.ConclusionsThis is a rare case of newly diagnosed CML presenting with diverse ocular manifestations in both eyes. The disparate outcomes in eyes with varying lesion stages underscore the importance of prompt diagnosis.

Neuropsychological manifestation as an initial presentation of pediatric chronic myeloid leukemia: A case report of diagnostic conundrum and review of literature

Neuropsychological manifestation as an initial presentation of pediatric chronic myeloid leukemia: A case report of diagnostic conundrum and review of literature

Rare presentation of chronic myeloid leukemia in a HIV patient: A coincidence or an association with a genetic basis? Review of literature

Rare presentation of chronic myeloid leukemia in a HIV patient: A coincidence or an association with a genetic basis? Review of literature

Chronic myeloid leukemia presenting with disc edema: A case report

The aim is to report a case of chronic myeloid leukemia (CML) in a 20-year-old female who initially presented with bilateral optic disc edema and headaches, and to raise awareness of uncommon causes of disc edema that could potentially be life-threatening. The patient first presented in June 2022 with blurry vision in the left eye, headache, and neck pain of 6 months duration. Initial examination revealed a young woman in no acute distress, with a blood pressure of 100/60 mm Hg and a body mass index of 26.98. Her pinhole visual acuity was 6/9 in both eyes, extraocular motility was full bilaterally, and both pupils were briskly reactive. However, she had bilateral optic disc edema. Both brain and orbital magnetic resonance imaging (MRI) revealed normal findings. Serological tests for syphilis and human immunodeficiency virus were negative. Based on these and the clinical features, an initial diagnosis of idiopathic intracranial hypertension was made, and the patient was started on acetazolamide tablets. She was followed up for approximately 1 year without resolution of symptoms. At a follow-up visit in December 2023, she was found to have developed dilated tortuous vessels with discrete retinal lesions. A peripheral blood film result showed that the patient had CML. In conclusion, a high index of suspicion should be entertained when managing a patient with unexplained bilateral disc swelling with normal brain MRI study. A complete blood workup, including a peripheral blood film, should be included as a part of the routine investigation of these patients so that diagnosis and treatment could be instituted early.

An Uncommon Presentation of Chronic Myeloid Leukemia: Osteolytic Bone Lesion as the Initial Manifestation

An Uncommon Presentation of Chronic Myeloid Leukemia: Osteolytic Bone Lesion as the Initial Manifestation

Chronic Myeloid Leukemia Presenting with Megakaryocytic Blast Crisis: a Case Report with a Favorable Outcome

Chronic myeloid leukemia (CML) belongs to the chronic myeloproliferative neoplasms, a clonal disorder of pluripotent hematopoietic stem cells, arising because of a hallmark genetic anomaly- reciprocal translocation between chromosomes 9 and 22, known as the Philadelphia chromosome, which generates the BCR::ABL fusion gene. The disease can have a biphasic/triphasic evolution, comprised of an accelerated phase and a terminal blast phase (usually biphasic when blastic phase is lymphoid in nature). Most cases present during the chronic phase of the disease, with only 10% of cases presenting in the blast phase. Megakaryocytic blast crisis is an unusual form of presentation, accounting for less than 3% of cases and it commonly carries an unfavorable prognosis compared to the classic myeloid, lymphoid or even mixed lineage blast crises. Aim: To bring forth an unusual case of CML presenting with megakaryocytic blast crisis and the therapeutic challenges associated with this particular presentation, unexpectedly ending with a favorable outcome. Matherial and methods: we hereby present the case of a 50 year old female patient, diagnosed in 2015 with CML- megakaryocytic blast crisis, who underwent treatment with a tyrosine kinase inhibitor (TKI)- imatinib and intensive chemotherapy regimen 3+7, without satisfactory response. A second induction chemotherapy regimen followed (MEC), alltogether with the same TKI and cytoreductive treatment (hydroxyurea/HU), but still without achieving a complete remission. Thus, the TKI was switched to dasatinib and complete normalisation of blood counts followed (including thrombocytes). However, there was still inadequate molecular and cytogenetic response. Therefore, TKI dosage was increased and after 4 months the patient achieved complete remission, with no detectable BCR::ABL transcript. She spent 9 years on the same TKI until important pleural effusion developed, which required switching to another 2nd generation TKI- bosutinib, based on the patients age, genetic profile and commorbidities. She is still in complete remission without detectable BCR::ABL transcript/Philadelphia chromosome to this day (2024). Results and conclusions: this is the case of a rare presentation of CMLmegakaryocytic blast crisis, who underwent both TKI and intensive chemotherapy induction regimens, marked by a favorable unexpected outcome.

Atypical presentation of chronic myeloid leukemia as extreme thrombocytosis

Chronic myeloid leukemia (CML) is a type of myeloproliferative neoplasm which typically presents with leukocytosis but rarely presents with extreme thrombocytosis with no/mild increase in leukocytes. Here, we describe a unique case of 42-year-old female with peripheral blood smear features that were favoring essential thrombocythemia (ET). However, further studies such as bone marrow and cytogenetic study were suggestive of CML in chronic phase. In some cases, CML mimics ET and cytogenetic study is the confirmatory test. ET and CML have different therapeutics and prognosis, and it is always necessary to consider CML as a differential diagnosis in cases of isolated thrombocytosis/extreme thrombocytosis cases.

An Unusual Case Report - An Adult of Chronic Myeloid Leukemia Presenting as Acute Myeloid Leukemia M7 (Megakaryocytic Blast Crisis)

An Unusual Case Report - An Adult of Chronic Myeloid Leukemia Presenting as Acute Myeloid Leukemia M7 (Megakaryocytic Blast Crisis)

Myeloid Sarcoma Masquerading as a Breast Lump: A Unique Presentation of Chronic Myeloid Leukaemia in Chronic Phase

Myeloid Sarcoma Masquerading as a Breast Lump: A Unique Presentation of Chronic Myeloid Leukaemia in Chronic Phase

Chronic Myeloid Leukemia Presenting with Mixed Phenotype B/Myeloid Blast Phase: A Diagnostic Challenge

Abstract Introduction/Objective Chronic myeloid leukemia (CML) is typically identified in chronic phase, however about 5% of cases present in a more advanced phase. In blast phase (BP), the blasts are most commonly of myeloid lineage, less commonly lymphoid, and rarely mixed lineage cases are encountered. A mixed phenotype at presentation raises the possibility of a de novo mixed phenotype acute leukemia (MPAL) with BCR::ABL1 fusion and requires careful correlation with ancillary studies to confirm the diagnosis. Methods/Case Report The patient is a 60-year-old man who presented with leukocytosis, anemia, normal platelet count, and blasts identified on peripheral blood smear examination. A bone marrow biopsy demonstrated hypercellular marrow and a dimorphic population of large and small blasts, comprising nearly 70% of cellularity. Flow cytometry demonstrated distinct myeloid and B-lymphoid blast populations, both of which expressed CD13 and TdT. Interphase fluorescence in situ hybridization (FISH) showed the BCR::ABL1 fusion in more than 90% of nuclei, including segmented nuclei, and conventional cytogenetic studies showed t(9;22)(q34.1;q11.2) in 19 of 20 metaphases. Molecular studies demonstrated the minor BCR::ABL1 p190 transcript, as well as ASXL1 and dual RUNX1 mutations. The discrepant number of BCR::ABL1 positive cells by FISH and conventional cytogenetic studies compared to the proportion of blasts, and its presence in segmented nuclei, enabled a diagnosis of CML with mixed phenotype blast phase despite absence of features such as basophilia or significant granulocytic left shift. Results (if a Case Study enter NA) NA Conclusion We share this case of CML in mixed phenotype blast phase to highlight the importance of careful evaluation in distinguishing this rare presentation from de novo MPAL, particularly with BCR::ABL1 fusion. Features such as a disproportionate number of nuclei or metaphases carrying the t(9;22) translocation on FISH or conventional cytogenetic studies and the presence of BCR::ABL1 in mature granulocytes can be helpful when other morphologic features of CML are absent.

Is the Prognosis of People with Chronic Myeloid Leukaemia Presenting in Accelerated Phase Always Worse Compared with People Presenting in Chronic Phase?

Background Some data, albeit controversial, suggest people with chronic myeloid leukaemia (CML) presenting in accelerated phase have the same prognosis as people with CML presenting in chronic phase but with high-risk co-variates. Objectives Interrogate the heterogeneity of subjects presenting in accelerated phase and compare their outcomes with persons presenting in high-risk chronic phase. Methods We interrogated data from 2132 consecutive subjects ≥ 18 years with CML presenting in accelerated (N = 274) or chronic phase (N = 1858). Subjects were seen at Peking University People's Hospital from January, 2006 to June, 2023. Criteria for accelerated phase according to the European LeukemiaNet (ELN) 2020 criteria included: ≥ 1 of the following: (1) blood or bone marrow blasts ≥ 15% but < 30%; (2) blood basophils ≥ 20%; and (3) platelet concentration < 100 × 10E + 9/L unrelated to therapy. Chronic phase was defined according to the ELN 2020 criteria. For subjects in chronic phase ELTS score at diagnosis was calculated and used to classify subjects into risk cohorts. Transformation was defined as blood or bone marrow blasts ≥ 30% during TKI-therapy. Transformation-free survival (TFS) was calculated as the interval from starting TKI-therapy to transformation, death or censored at a transplant or last follow-up. We then compared therapy responses and outcomes between defined accelerated phase cohorts and chronic phase risk cohorts using competing risk models and Kaplan-Meier survival analyses. Cox regression analyses were used to further explore the co-variates associated with therapy responses and outcomes. Results Amongst the 274 subjects presenting in accelerated phase 205 (75%), 39 (14%) and 31 (11%) were classified based on basophilia, excess blasts or thrombocytopenia. Amongst the 1858 subjects presenting in chronic phase 1220 (66%), 461 (25%) and 176 (9%) were ELTS low-, intermediate- and high-risk. In multi-variable analyses we use chronic phase subjects in ELTS low-, intermediate- and high-risk cohort as reference. Subjects in accelerated phase with basophilia had better therapy responses (MMR, HR = 1.2 [1.0, 1.5], p = 0.05; MR 4, HR = 1.7 [1.3, 2.2], p < 0.001; MR 4.5, HR = 2.0 [1.5, 2.6], p < 0.001) and similar TFS and survival (TFS, HR = 0.9 [0.5, 1.6], p = 0.60; survival, HR = 1.4 [0.6, 3.2], p = 0.45) compared with the ELTS intermediate-risk cohort. Accelerated phase subjects with excess blasts and with thrombocytopenia therapy responses like chronic phase subjects with the ELTS intermediate- and high-risk cohorts. TFS and survival were similar with ELTS high-risk cohort. These comparisons held in subjects receiving initial imatinib-therapy but were less clear in subjects receiving initial 2 nd generation TKI therapy. Similar findings were also obtained using the WHO 2016 criteria. Conclusions Subjects presenting in accelerated phase defined by basophilia had better therapy responses but similar TFS and survival compared with subjects presenting in chronic phase in the ELTS intermediate-risk cohort. Subjects presenting in accelerated phase with excess blasts or thrombocytopenia had comparable therapy responses compared with subjects presenting in chronic phase in the ELTS intermediate- and high-risk cohorts whereas their TFS and survival were like those or worse compared with subjects presenting in chronic phase in the ELTS high-risk cohort. The implication of our findings is presenting in accelerated phase per se does not always confer a worse prognosis compared with subjects presenting in chronic phase.

Atypical presentation of chronic myeloid leukemia with e13a2 BCR-ABL1 fusion transcript in a young male

Chronic myeloid leukemia (CML) is usually a disease of the elderly population characterized by a clonal hematopoietic progenitor cell disorder propelled by the product of the breakpoint cluster region-Abelson murine leukemia (BCR-ABL) chimeric gene, known as the Philadelphia chromosome. It occurs due to a balanced reciprocal translocation between the long arms of chromosomes 9 and 22, which codes for tyrosine kinase. The incidence of CML increases with age, and the median age of diagnosis in Western countries is 64 years. The most common finding at presentation is abdominal pain due to splenomegaly which is present in about half of the patients. We present a rare case of CML in the chronic phase where a young 23-year-old male presented with complaints of severe bilateral lower limb bone pain restricting mobility at the hip joint for 1 year. The upper end of the femur showed heterogeneous areas of bone infarct on the technetium 99 m-methylene diphosphonate bone scan. He was treated with imatinib mesylate, and he entered remission within 5 months of starting therapy. This case highlights the atypical presentation of the chronic phase of CML with the e13a2 BCR-ABL fusion transcript in a young patient and the complete recovery achieved with standard therapy.

Pediatric chronic myeloid leukemia (CML) presenting with ocular manifestations: A case report

A 14-year-old male child presented to the ophthalmologist with complaints of floaters and headache. Ophthalmic examination revealed dilated and tortuous retinal veins, perivascular infiltrates scattered throughout the fundus, and multiple white centered hemorrhages (leukemic infiltrates). A provisional diagnosis of leukemia or lymphoma was made. The diagnosis was confirmed by further systemic workup and complete blood count. A final diagnosis of CML in chronic phase was made after bone marrow aspiration, biopsy, and presence of BCR-ABL translocation. This case report highlights the role of an ophthalmologist in diagnosing and prognosticating a rare entity of pediatric CML with ocular manifestation as its presenting symptoms.

Ocular involvement—An unusual initial presentation of chronic myeloid leukemia: A case report

ABSTRACT Chronic myeloid leukemia (CML) patients frequently exhibit systemic symptoms such as fatigue, abdominal discomfort, weight loss, and fever but rarely can have atypical initial presentation in the form of ophthalmic manifestations, which can precede the diagnosis of the primary malignancy. We describe a case of a 29-year-old male who presented in our ophthalmology out-patient department (OPD) with complaints of painless, diminution of vision, which was sudden in onset in right eye (RE) and loss of vision in left eye (LE) for four and seven days, respectively. There had been a history of loss of weight and appetite for the past 2 months. The visual acuity (VA) recorded was finger counting two meters in RE and perception of light in LE with an inaccurate projection of rays in both eyes (BE). The anterior segment evaluation of both eyes (BE) was normal. Fundus revealed multiple elevated yellow subretinal lesions with exudative detachment in the RE and no view in the LE. Ultrasound-Brightness (USG B) scan in the LE revealed multiple hyperreflective echoes likely vitreous hemorrhage. Optical coherence tomography (OCT) showed subretinal hyperreflectivity with surrounding edema in RE suggestive of leukemic infiltrates. On further systemic investigations, chronic myeloid leukemia-chronic phase (CML-CP) was detected; hence, the diagnosis of RE exudative retinal detachment (RD) and LE vitreous hemorrhage with CML-CP was made. Ophthalmic involvement is more often seen in acute than chronic leukemia, which makes the diagnosis challenging. We describe a unique case of a young patient with CML-CP who initially presented with ocular involvement preceding systemic diagnosis. This case report illustrates the importance of a primary care physician or an ophthalmologist in the early diagnosis and prompt management of hematological malignancy, as ophthalmic manifestations may be a rare initial presenting feature in CML-CP. These conditions require urgent referral to a hematologist by a primary care physician in the view of early commencement of therapy.

Personalized Management of Chronic Myeloid Leukemia Presenting with Ovarian Apoplexy and Review of Health Emergency Cases

Introduction: Chronic myeloid leukemia (CML) may significantly affect quality of life and life expectancy in the accelerated and acute phases, especially if presented with health emergencies. Objective: The purpose of the study was the evaluation of the personalized management milestones in CML cases presented with ovarian apoplexy and the analysis of the international experience on health emergencies in this leukemia. Materials and Methods: An original, case-report study was performed. The diagnosis was confirmed by cytological, cytogenetic and molecular examinations of the peripheral blood and bone marrow at the comprehensive research cancer center— Institute of Oncology from Moldova. The real-time PCR was performed for quantitative detection of BCR-ABL gene p210 and p190 transcripts. The ECOG Scale and complete hematologic response (CR) estimated the short-term results. The ECOG performance status served as a measure of functional status. Its scores range from 0 to 5 and correlate with the level of patient functioning. CR is the disappearance of all detectable clinical and hematological signs of malignant neoplasm in response to treatment. CR span was assessed in months. The overall and relapse-free survivals asserted the long-term results of treatment, and were evaluated in months as a case. Results: CML may be manifested by life-threatening conditions, including infections and thrombotic events, splenic infarcts and ruptures, bleedings, etc. CML with the uncommon onset under the form of the ovarian apoplexy is described. The hematological CR was obtained under the treatment with imatinib mesylate. The molecular CR and the long-lasting overall survival (197.5 months) were achieved after the treatment with nilotinib. Under the treatment with tyrosine kinase inhibitors, the patient did not experience the disease burden and side effects, and resumed her work, with a good life quality (ECOG score is 0). Discussion: CML may be manifested by life-threatening health emergencies, especially thrombotic events, splenic infarcts and ruptures, bleedings, etc. The CML patients with high leukocyte and platelet counts may experience also different symptoms of hyperviscosity: tinnitus, priapism, stupor, visual abnormalities and cerebrovascular accidents. However, the management of CML with the life-threatening conditions, especially in the accelerated and acute phases, should be realized with participation of the multidisciplinary teams. Conclusions: The ovarian apoplexy may serve as a presenting feature of CML in young females with a highly elevated leukocyte count. The reported CML case underwent successful personalized management with 2 generations of tyrosine kinase inhibitors, even if designated with Socal intermediate-risk score and complicated by a health emergency.

Chronic myeloid leukaemia with generalized lymphadenopathy – a high risk category

Chronic myeloid leukaemia (CML) presenting with generalized lymphadenopathy is an uncommon manifestation of CML. Lymphadenopathy in these patients could be due to extramedullary involvement by myeloid cells with all stages of maturation, blast infiltration, co-existing lymphoproliferative disorder or reactive lymphadenopathy. We report a case of a 38-year-old male with CML in chronic phase and generalized lymphadenopathy at diagnosis. He transformed to blast crisis within 14 weeks of diagnosis despite tyrosine kinase inhibitor (TKI) therapy. This case indicates that CML patients with lymphadenopathy are at high risk of disease progression and early blast transformation and therefore candidates for aggressive treatment.

Is the 2nd Generation Tyrosine Kinase-Inhibitor a Better Initial Therapy Than Imatinib in Persons with Chronic Myeloid Leukemia Presenting in Accelerated Phase: A Multicenter Retrospective Study

Objectives Compare the treatment responses and outcomes between persons with chronic myeloid leukemia (CML) presenting in accelerated phase receiving a 2nd-generation tyrosine kinase inhibitor (2G-TKI) and imatinib as initial therapy. Methods Data from 442 consecutive subjects with CML presenting in accelerated phase according to the European LeukemiaNet (ELN) 2020 criteria from 32 centers were retrospectively collected. Propensity score matching (PSM) was performed to adjust for differences in baseline co-variates between subjects receiving a 2G-TKI or imatinib as initial therapy. Cumulative incidences of CCyR, MMR and MR4.5 were calculated using the Fine-Gray model. Transformation was defined as blood or bone marrow blasts ≥ 30% during TKI-therapy. Transformation-free survival (TFS) and survival were calculated by the Kaplan-Meier model. Results 442 subjects with CML presenting in accelerated phase were reviewed, including 147 (33%) received 2G-TKI and 295 (67%) received imatinib as initial therapy. 270 (61%) were male, median age was 43 years (Interquartile range [IQR], 31-55 years), median follow-up was 28 months (IQR, 13 - 53 months) in the 2G-TKI cohort and 51 months (IQR, 25 - 94 months) in the imatinib cohort reflecting the later availability of 2G-TKIs (p < 0.001), median treatment duration was 19 months (IQR, 9 - 41 months) in the 2G-TKI cohort and 32 months (IQR, 12 - 68 months) in the imatinib cohort (p < 0.001). 28 (26%) receiving 2G-TKI and 100 (34%) subjects receiving imatinib switched to another TKI during treatment. 379 subjects were identified by a 2:1 propensity-score matching in the 2G-TKI (n = 139; 37%) and imatinib (n = 240; 63%) cohorts. In the 2G-TKI and imatinib cohorts, the cumulative incidence of 2-year CCyR was 87% (95% Confidential Interval [CI], 86%, 87%) vs. 84% (84%, 84%, p < 0.001); MMR, 83% (82%, 83%) vs. 65% (65%, 65%, p < 0.001); MR4.5, 43% (42%, 44%) vs. 29% (28%, 29%, p = 0.006) when switching to another TKI, a transplantation and death were considered as competing events. When switching to another TKI was not considered as a competing event, cumulative incidences of 2-year CCyR were 91% (91%, 92%) vs. 83% (83%, 83%, p < 0.001); MMR, 80% (80%, 81%) vs. 66% (66%, 66%, p = 0.002); MR4.5, 40% (40%, 41%) vs. 28% (27%, 28%, p = 0.005) in the 2G-TKI and imatinib cohorts. The 2-year probabilities of TFS were 88% (81%, 94%) vs. 92% (88%, 96%, p = 0.116); survival, 97% (94%, 100%) vs. 98% (96%, 100%, p = 0.530) in the 2G-TKI and imatinib cohorts when censored at switching to another TKI, a transplantation or at last follow-up. Similarly, 2-year probabilities of TFS were 94% (89%, 98%) vs. 93% (90%, 96%, p = 0.868); survival, 97% (94%, 100%) vs. 96% (93%, 98%, p = 0.562) in the 2G-TKI and imatinib cohorts when censored at a transplantation or at last follow-up. Conclusions Persons with CML presenting in accelerated phase receiving a 2G-TKI as initial therapy achieved higher cumulative incidences of MMR and MR4.5 than those receiving imatinib, but similar TFS and survival. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Hematoma or Bleeding As Initial Presentation of Chronic Myeloid Leukemia (CML): Review

Background: Chronic myeloid leukemia (CML) is defined per the WHO 2016 classification as a myeloproliferative neoplasm characterized by the chromosomal translocation t (9; 22) (q34.1; q11.2). Patients with CML can have a very wide range of symptoms at presentation. Most patients presents as an incidental finding on a routine complete blood count (CBC). Symptoms might include B symptoms like fever, loss of weight, and night sweats. While splenomegaly is the most common initial presentation in the chronic phase, in accelerated or blast phase of CML, the presentations might resemble acute leukemia with signs and symptoms of anemia, bleeding, petechiae, and ecchymosis. Bleeding might be the initial presentation of acute leukemia; however, it is scarce for CML, especially the chronic phase, to present initially with spontaneous bleeding without coagulopathy and thrombocytopenia. The mechanism behind this rare perennation is not known up to the moment. Methods: A literature search was performed for English language articles using Medline data through PubMed, Scopus, and Google Scholar, for any date up to June 2022. The following keywords were used "CML," "spontaneous bleeding," "bleeding," and "hematoma.". We did not find any retrospective, prospective or clinical trials or reviews on this rare topic. We decided to review all the case reports and case series published in the literature and collect all the general characteristics, common findings, treatment, and outcomes Results: Thirty articles with 30 patients were included. Most of them were males, 23 (76.7%) with a median age was 40 years, and the majority were of Asian origin 13 out of 17 (76.4%). Hematoma sites were different; intracranial was most affected in 11 (36.6%) patients, followed by intraabdominal and musculoskeletal (MSK) bleeding in 9 (30%) patients each. One case was reported as an intrathoracic bleed. All the patients diagnosed with CML chronic phase except one patient in accelerated phase without thrombocytopenia and one presented with blast crisis. Coagulopathy was reported in 2 patients only. The average duration of symptoms was around 6 days, and the median white blood cell count on presentation was 232,000, while 192,000 for platelets and 8.5 gm/dL for hemoglobin. The sequel reported were bilateral deafness in 1 patient, unilateral blindness in 1 patient, and one admission to the intensive care unit. Sixteen (53%) patients received imatinib 400 mg daily. Surgery was done in 12 (40%) patients, and a similar number were treated conservatively. Documented recovery was reported in 10 (33%) patients. Overall survival was reported in 26 patients. 20 (66.6%) were alive, while 6 (33.3%) were dead. The cause of death was reported to be intracranial hemorrhage in 2 cases, sepsis in one case, and splenic rupture in one case. Conclusion: Spontaneous bleeding or hematoma as an initial presentation of CML is rare. Thrombocytopenia and coagulopathy do not explain most of the cases. We hypothesize that male sex, Asian ethnicity, and hyperleukocytosis could be a risk factors. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Prevalence of anemia at diagnosis of pediatric chronic myeloid leukemia and prognostic impact on the disease course.

The clinical presentation of chronic myeloid leukemia (CML) at diagnosis differs in children compared to adults. At younger age, anemia appears to be frequent at diagnosis, but its prevalence and its impact on prognosis are not well known. In the International Registry of Childhood CML, we selected children and adolescents in chronic phase at diagnosis of CML and treated upfront with imatinib. We examined their hemoglobin level at diagnosis according to the WHO grades to assess the prevalence of anemia and its impact on response to tyrosine kinase inhibitors (TKIs). Data on 430 patients were included. Anemia at diagnosis was observed in 350 patients (81%), with a mean hemoglobin level of 96.4g/l (SD 23.6). Among them, 182 patients (52%) presented with moderate anemia and 110 (31%) with severe anemia while 58 (17%) had mild anemia. Compared with mild and no anemia, moderate and severe forms were significantly associated with younger age at diagnosis, asthenia, splenomegaly, and increased leukocyte and basophil counts. Delays in achieving major and deep molecular responses were significantly increased for patients with moderate and severe anemia, and also failure of imatinib treatment was more frequent in these two sub-cohorts. However, hemoglobin level was not significantly associated with survival. Anemia at diagnosis of pediatric CML was frequent and may be considered as a prognostic factor.

P722: IS THE SECOND-GENERATION TYROSINE KINASE INHIBITOR A BETTER THERAPY THAN IMATINIB FOR PERSONS WITH CHRONIC MYELOID LEUKEMIA PRESENTING IN ACCELERATED-PHASE?

Background: Although most patients with chronic myeloid leukemia (CML) were presenting in the chronic phase at diagnosis, approximately 5% presented with features of accelerated phase. The features of accelerated phase were variably defined by different criteria, such as MD Anderson and European LeukemiaNet (ELN) criteria most often used. Regardless of these criteria used to defined accelerated phase, only scant data were available regarding the prognostic co-variates for outcome. Moreover, evidence that patients could benefit more from the second generation (2G-) tyrosine kinase inhibitor (TKI) than imatinib as initial therapy was still extremely limited. Aims: Explore whether patients with chronic myeloid leukemia (CML) presenting in accelerated-phase could benefit from initial second-generation tyrosine kinase inhibitor (2G-TKI) therapy. Methods: We interrogated data from consecutive patients with CML presenting in accelerated phase at diagnosis by the MD Anderson criteria (n = 325) or ELN criteria (n = 275). Propensity score matching (PSM) analysis was used to compare cytogenetic and molecular responses and outcomes in those receiving initial imatinib versus a 2G-TKI. COX multi-variable regression model was performed to identify the prognostic co-variates. Results: In the cohort defined by MD Anderson criteria, PSM analysis showed that patients receiving initial 2G-TKI (n = 89) had higher probability of achieving a CCyR (p = 0.01), MMR (p = 0.04) and MR4.5 (p = 0.004) than those receiving imatinib (n = 156), however, probabilities of FFS, PFS and survival were similar. Multi-variable analyses showed that lower platelets and higher blasts in blood or bone marrow were significantly-associated with poor FFS, PFS and/or survival (Figure A). In an attempt to determine whether choice of therapy contributed to the differences in responses and outcomes, platelets < 600 × 10E+9/L and blood / bone marrow blasts ≥ 5% were identified as the prognostic categorical co-variates by ROC analyses. The entire cohort were classified into low- (possessing none of these co-variates, n = 57), intermediate (possessing any 1 of these co-variates, n = 127) or high-risk (possessing all of these co-variates, n = 61) subgroups. There were significant differences in probabilities of outcomes among these risk subgroups (all p values < 0.001, Figure B). In each risk subgroup, patients receiving initial 2G-TKI had the similar outcomes to those receiving imatinib except higher incidences of responses including CCyR, MMR and MR4.5. The same results were observed in the cohort defined by ELN criteria. Image:Summary/Conclusion: Initial 2G-TKI lead to similar outcomes as imatinib in patients with CML presenting in accelerated-phase except higher therapy responses.