Waldenström macroglobulinaemia (WM) is an incurable chronic B‐cell malignancy, but highly responsive to treatment. Treatments include fixed‐duration chemotherapy and continuous oral chemoimmunotherapy. In this expanding field, it is important to have reliable information on the impact of the various therapies on patients’ quality of life (QoL). Patient reported outcome measures (PROMs) are increasingly recognised as important to understand patient experience of disease beyond traditional clinical outcome measures. Four QoL questionnaires (EORTC QLQ‐C30 [European Organisation for Research and Treatment of Cancer quality of life core questionnaire], BIPQ [Brief Illness Perception Questionnaire], HADS [Hospital Anxiety and Depression Scale], EQ‐5D‐5L [EuroQoL 5‐dimensional descriptive system questionnaire]) are embedded in the UK national WM registry, the Rory Morrison Registry. We reviewed the results from a snapshot of PROMs. As of November 2021, 155 patients completed PROM data with 98% completion rate across all 58 questions. Complete clinical information was available for 52 patients. The majority of QoL questions (69%) failed to elicit a notable median response. Only four questions elicited statistically significant responses when comparing groups, and these were exclusively found in the EuroQoL‐5D‐5L and HADS questionnaires. Our data suggest that widely used questionnaires may not be suitable for patients with WM. We advocate the development of WM‐specific outcome measures to overcome this.

Waldenström Macroglobulinaemia is an indolent disease which is recognised as contributing to a diverse range of disabling symptoms which can impact quality of life. Little data are available in evaluating patient and healthcare professional views on quality-of-life questionnaires (QLQ) used to evaluate quality of life in Waldenström Macroglobulinaemia. We aimed to assess patient perception of their disease and the relevance of the currently employed QLQs in practice by patients and healthcare professionals. One-hundred-and-twenty patients (22% treatment-naïve) and thirty-four healthcare professionals completed an international survey to understand their perception of the relevance of the currently employed QLQ. Out of 415 questions, 257 questions were classified by patients as relevant. Five themes were discerned as relevant: fatigue, mobility, dyspnoea, paraesthesia, pain in the hands and feet, sleep (physical health limitations); contentment (psychological impact); social and family support. The following were considered irrelevant: lumps or swelling, dry mouth, trouble hearing, sexual dysfunction (physical symptoms); post-traumatic stress (psychological impact); education and training and financial support. Healthcare professionals identified EORTC QLQ-C30 as the most commonly used instruments for Waldenström Macroglobulinaemia trials.

It is widely assumed that the word hemophilia is derived from the two Greek words, “hema” meaning “blood” and “philia” meaning “the love of.” However, hemophilia treaters and persons with hemophilia might have often wondered how “love of blood” could stand for the increased tendency to bleed in this hereditary condition. What made the Greeks choose hemophilia when naming this bleeding disorder? Or did they name it at all? Although symptoms associated with hemophilia have been known for centuries, most experts refer to the 19th century as the period when it was described as a separate disease entity.[1] [2] [3] In the first decade of the 1800s, the American physician, John Conrad Otto published his observations of two families, the Smith family from Plymouth neighborhood and the Benjamin Benney family from Maryland, who presented with bleeding symptoms.[4] He noticed the females in these families transmitted the hemorrhagic disease while the males were affected and he called these males “bleeders.” As might happen with any new disease, several names were proposed to describe it. In the older French books of the time, hemophilia was called as diathese hemorrhagique (hemorrhagic diathesis), hemorrhagie constitutionnelle (constitutional hemorrhage), and purpura constitutionnelle (constitutional purpura).[5] The patients themselves were called hommes saignants (bleeding males) similar to the “bleeders” terminology of Otto. The German choice of words for hemophilia were Bluterkrankheit (bleeder illness) commonly, and Blutsucht and Blutungssucht (blood addiction) less commonly, Bluter being the German translation of bleeder.[5] What is noticeable so far is the absence of descriptions of hemophilia in the 19th century Greek literature. Indeed, Greek dictionaries explain hemophilia as a Greek work constructed outside of Greece.[6] Historical accounts credit a German physician, Friedrich Hopff, working in the first half of the 19^th century as the inventor of the word “hemophilia.”[1] [2] [3] There was a sudden surge in interest in the study of bleeding disorders during this period, possibly because bleeding symptoms were noted in several members of the Royal family. The most celebrated physician of the time, Johann Lukas Schoenlein (his name is remembered in the bleeding disorder, Henoch Schoenlein purpura) was keen on a developing a classification system for pathological conditions according to their characteristics and symptoms.[7] He created the three classes—morphae, hematoses, and neuroses—which were subdivided into families, and further into groups similar to the classification schemes known in botany and zoology.[7] It is certainly possible that some of the patients included in the class of hematoses would have had hemophilia, although Schoenlein had a high disinterest in publishing his findings. He is said to have called the disease “hemorrhaphilia” (love of bleeding) in his Vorlesungen (lectures).[5] The word “hemophilia” was the apparent invention of Schoenlein's student, Friedrich Hopff, who used it in the title of his dissertation, “Über die Hämophilie oder die erbliche Anlage zu tödtlichen Blutungen” (“about hemophilia or the hereditary predisposition to fatal bleeding”).[5] Why may he have chosen this particular word? Did he have the hereditariness of the bleeding disorder in mind? While using the Greek terminology, the exact spelling of “philia” makes a significant difference to the meaning. If spelt with a Greek iota as ϕιλία where the accent is on the penultimate syllable, it would mean affinity toward blood as “filia,” the ancient Greek word for friendship. However, if spelt with a Greek ypsilon as ϕυλή (fyli) where the accent is on the final syllable, this means race or tribe. Since the title of Hopff's dissertation included hereditary, it is possible that he was intending ϕυλή to indicate the “inheritedness” of hemophilia. Wickham Legg's treatise on haemophilia[5] gives a pointer to this possibility: “The etymology of the word seems to be plain αἷμα and ϕιλία. M. de Fleury, however, prefers to derive the name from ϕυλή, a race, or tribe, with reference, I suppose, to its hereditary character.” We sought the assistance of a Greek linguist (acknowledged) for a possible explanation for Hopff's choice of the word “hemophilia.” He suggested an alternate meaning of the word “haima” to be “blood flow” rather than just blood. Thus, hemophilia would have possibly meant for Hopff, the “love of blood flow.” Did he choose “hemophilia” rather than “hemorrhophilia” as the title of his thesis because he wanted to distinguish himself from his eminent professor? Did he want to be more specific about the hereditary nature of the disorder? Do we wrongly assume that it stands for the love of blood instead of the love of blood flow? In summary, due to a quirk of language, the word “hemophilia” has come to commonly mean “love of blood” when it may have instead denoted a hereditary tendency to bleeding or “the love of blood flow” at the baptismal font. Publication Date: 24 August 2021 (online) © 2021. Thieme. All rights reserved. Thieme Medical Publishers, Inc. 333 Seventh Avenue, 18th Floor, New York, NY 10001, USA