Michael Steurer

Summary Immune thrombocytopenia (ITP) is an acquired autoimmune phenomenon resulting in low platelet count and increased bleeding risk. Goals of upfront management include prompt control of severe bleeding—which is rare—as well as induction and maintenance of a hemostatic platelet count. Thus, optimal management of ITP patients is often challenging and requires a highly individualized approach. Many patients may not suffer significant bleeding despite severe thrombocytopenia and the risk of toxicity associated with treatment may outweigh its benefit. Most patients treated with standard first-line regimen of glucocorticoids achieve an initial response. However, the rate of long-term remission remains low and multiple lines of therapy are often required. Current investigations aim at defining the subgroup of patients at risk of relapse and providing intensified risk-balanced induction regimens to improve long-term disease control. This short review summarizes current and emerging treatment strategies in adult ITP.

BACKGROUND AND PURPOSE: Dose reduction on CT scans for surgical planning and postoperative evaluation of midface and orbital fractures is an important concern. The purpose of this study was to evaluate the variability of various low-dose and iterative reconstruction techniques on the visualization of orbital soft tissues. MATERIALS AND METHODS: Contrast-to-noise ratios of the optic nerve and inferior rectus muscle and subjective scores of a human cadaver were calculated from CT with a reference dose protocol (CT dose index volume = 36.69 mGy) and a subsequent series of low-dose protocols (LDPs I–4: CT dose index volume = 4.18, 2.64, 0.99, and 0.53 mGy) with filtered back-projection (FBP) and adaptive statistical iterative reconstruction (ASIR)-50, ASIR-100, and model-based iterative reconstruction. The Dunn Multiple Comparison Test was used to compare each combination of protocols (α = .05). RESULTS: Compared with the reference dose protocol with FBP, the following statistically significant differences in contrast-to-noise ratios were shown (all, P ≤ .012) for the following: 1) optic nerve: LDP-I with FBP; LDP-II with FBP and ASIR-50; LDP-III with FBP, ASIR-50, and ASIR-100; and LDP-IV with FBP, ASIR-50, and ASIR-100; and 2) inferior rectus muscle: LDP-II with FBP, LDP-III with FBP and ASIR-50, and LDP-IV with FBP, ASIR-50, and ASIR-100. Model-based iterative reconstruction showed the best contrast-to-noise ratio in all images and provided similar subjective scores for LDP-II. ASIR-50 had no remarkable effect, and ASIR-100, a small effect on subjective scores. CONCLUSIONS: Compared with a reference dose protocol with FBP, model-based iterative reconstruction may show similar diagnostic visibility of orbital soft tissues at a CT dose index volume of 2.64 mGy. Low-dose technology and iterative reconstruction technology may redefine current reference dose levels in maxillofacial CT.

Objective: Romiplostim has maintained long‐term platelet counts in patients with immune thrombocytopenia (ITP) for up to 5 yr in clinical studies. This prospective observational study aimed to describe romiplostim utilisation and outcomes in European clinical practice.Methods: Adults with primary ITP who received romiplostim in routine care were eligible.Results: Three‐hundred and forty patients were eligible for analysis, of whom 299 (88%) completed the 2‐yr observation period. The median age was 62 yr, with 43% of patients aged ≥65 yr, and two‐thirds of patients initiated romiplostim before splenectomy. The median average weekly dose of romiplostim was 2.8 μg/kg. The median baseline platelet count was 20 × 10⁹/L, which increased after 2 wk of romiplostim treatment and remained >50 × 10⁹/L thereafter. After romiplostim initiation, there was a decrease in rates of grade ≥3 bleeding events (from 12 to 2 per 100 patient‐years) and ITP‐related hospitalisations (from 87 to 33 per 100 patient‐years). The rate of thrombotic events was 2 per 100 patient‐years, and bone marrow fibrosis occurred in two patients.Conclusions: Romiplostim dosing, effectiveness and safety in an unselected real‐world ITP population seemed comparable with that observed in clinical studies.

Bendamustine is an established treatment option in chronic lymphocytic leukemia (CLL) and frequently used in Austria and Italy. Therefore, we analyzed 100 unselected, consecutive patients with CLL (treatment-naïve and relapsed/refractory) receiving bendamustine in a real-life setting. Most patients were treated with bendamustine in combination with rituximab (BR). However, bendamustine monotherapy was additionally evaluated. Patients treated with BR had a significantly higher overall response rate of 76% (complete response=22%) when compared to those treated solely with bendamustine (overall response rate=50%; complete response=13%). Overall survival (OS) and progression -ree survival (PFS) were significantly lower in the bendamustine-treated group (OS=14.3 months; PFS=8.3 months) compared to the BR group (OS=42.7; PFS=22.5 months; both p<0.001). In multivariate analysis, patients with a good cytogenetic risk and those receiving BR had a significantly better OS. Grade 3/4 hematological complications were seen in 32% of the patients. Hence, bendamustine, especially in combination with rituximab, is an effective therapy with manageable toxicity for non-selected patients with CLL including those pre-treated with fludarabine and the elderly.

Mast cell leukemia (MCL) is a rare, life-threatening malignancy defined by a substantial increase in neoplastic mast cells (MCs) in bone marrow (BM) smears, drug-resistance, and a poor prognosis. In most patients, the survival time is less than 1 year. However, exceptional cases may present with a less malignant course. We report on a 49-year-old female patient with MCL diagnosed in 2013. In February 2013, first symptoms, including flushing, headache, and diarrhea, were recorded. In addition, mild anemia was detected. The disease was characterized by a massive increase in well-granulated, mature, and often spindle-shaped MCs (80 %) in BM smears. The serum tryptase level amounted to 332 ng/mL. Like in most other MCL patients, no skin lesions were detected. However, unlike in other patients, tryptase levels remained stable, and no other signs or symptoms of MCL-induced organ damage were found. Sequencing studies revealed an isolated S476I point mutation in KIT but no mutation in codon 816. The patient received histamine receptor blockers but refused cytoreductive therapy. After 9 months, still no progression or organ damage was detected. However, progression with transformation to acute MCL occurred after 12 months. We propose that the chronic type of MCL with stable conditions, absence of organ damage, and a mature MC morphology is recognized as a distinct entity that should be distinguished from the acute variant of MCL.